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Every FDA drug approval from 2023: Full list of approved new drugs

After kicking off 2023 with a potential megablockbuster in Eisai and Biogen’s new Alzheimer’s drug Leqembi, in totality the FDA signed off on 10 more…



After kicking off 2023 with a potential megablockbuster in Eisai and Biogen’s new Alzheimer’s drug Leqembi, in totality the FDA signed off on 10 more novel drug approvals in 2023 when compared to last year.

The 57 novel drugs approved in 2023 (not counting gene therapies or vaccines) are the most since 2018 — the year of Gilead’s blockbuster HIV drug Biktarvy, which generated $10.4 billion in 2022 sales.

But peak 2023, as in 2022, came with approvals of new cell and gene therapies. In the case of 2023, the two approvals on Dec. 8 were for new sickle cell gene therapies, including Vertex and CRISPR Therapeutics’ Casgevy, the first CRISPR-based treatment in the US. Worth watching in 2024: how bluebird’s decision to price Lyfgenia nearly $1 million more than Casgevy will play out.

And the $2 million-plus gene therapies are likely to further test the bounds of the commercial markets in the US and Europe, where these potential cures have so far yet to muster wider coverage or access. With the backing of CBER’s chief Peter Marks, however, be prepared to see more of the same with respect to cell and gene therapy approvals in the coming years.

What to watch for in 2024: CMS will disclose its negotiated prices on Sept. 1 for the first 10 Part D drugs (not kicking in until 2026), and all eyes will be on CMS’ disclosure of net prices, with the investment bank Leerink Partners noting that biopharma companies may push back as net prices are not commonly disclosed publicly. — Zachary Brennan

  • Leqembi
  • Brenzavvy
  • Orserdu
  • Jaypirca
  • Jesduvroq
  • Lamzede
  • Filspari
  • Altuviiio
  • Skyclarys
  • Zavzpret
  • Daybue
  • Rezzayo
  • Zynyz
  • Joenja
  • Qalsody
  • Vowst
  • Arexvy
  • Elfabrio
  • Veozah
  • Miebo
  • Epkinly
  • Vyjuvek
  • Xacduro
  • Posluma
  • Paxlovid
  • Inpefa
  • Abrysvo
  • Columvi
  • Elevidys
  • Litfulo
  • Rystiggo
  • Ngenla
  • Lantidra
  • Roctavian
  • Beyfortus
  • Cyfendus
  • Vanflyta
  • Balfaxar
  • Xdemvy
  • Izervay
  • Zurzuvae
  • Talvey
  • Elrexfio
  • Sohonos
  • Veopoz
  • Aphexda
  • Ojjaara
  • Exxua
  • Pombiliti
  • Rivfloza
  • Velsipity
  • Bimzelx
  • Zilbrysq
  • Omvoh
  • Agamree
  • Loqtorzi
  • Fruzaqla
  • Defencath
  • Augtyro
  • Truqap
  • Ryzneuta
  • Ogsiveo
  • Fabhalta
  • Casgevy
  • Lyfgenia
  • Filsuvez
  • Wainua

  • Company: Eisai and Biogen
  • Price: $26,500 annually
  • Peak sales estimate: $7 billion annually, according to Eisai
  • Approval date: 01/06/2023

1. Leqembi

Active ingredient: lecanemab-irmb

Indication: To treat Alzheimer’s disease

Snapshot: Adherents of the amyloid hypothesis were vindicated in July when Leqembi became the first drug in 20 years to win a full-throated FDA approval for Alzheimer’s disease. By targeting amyloid proteins in the brains of people in the early stages of the disease, an 18-month course of the antibody infusion, given every two weeks, slowed cognitive decline by 27%. A weekly injectable form could come in 2024, along with a competing antibody from Eli Lilly. With their modest effects and the possible risks of brain bleeding and swelling, it’s still unclear how many patients will get the drugs, but Eisai hopes global sales of Leqembi could reach $7 billion by 2030. — Ryan Cross

  • Company: TheracosBio 
  • Price: $47.85 plus shipping for a 30-day supply through Mark Cuban’s Cost Plus Drugs
  • Peak sales estimate: N/A
  • Approval date: 01/23/2023

2. Brenzavvy

Active ingredient: bexagliflozin

Indication: To improve glycemic control in adults with type 2 diabetes mellitus as an adjunct to diet and exercise

Snapshot: SGLT2 inhibitor Brenzavvy was approved in January, but by July, it had already joined Mark Cuban’s Cost Plus Drugs plan for exclusive distribution. TheracosBio, founded in 2000 with a mission to bring low-cost drugs to market, reached out to Cuban’s company shortly after it formed in 2022 with the idea to potentially partner and compete against drugs like Invokana, Farxiga and Jardiance. Its approval was based on Phase III studies that showed reduced A1C and fasting blood sugar after 24 weeks of use. Additional benefits shown in the trials, although not approved indications, included cardiovascular risk reductions, weight loss and blood pressure reduction. — Beth Snyder Bulik

  • Company: Menarini Group
  • Price: $21,000 per month
  • Peak sales estimate: N/A
  • Approval date: 01/27/2023

3. Orserdu

Active ingredient: elacestrant

Indication: To treat estrogen receptor-positive, human epidermal growth factor receptor 2-negative, ESR1-mutated, advanced or metastatic breast cancer with disease progression following at least one line of endocrine therapy

Snapshot: The Menarini Group and Radius Health got their oral SERD drug Orserdu, or elacestrant, approved in January. It’s a drug class that has flummoxed some of the biggest names in Big Pharma, with Sanofi and Roche running into R&D setbacks in the last several years. Menarini bought the drug in 2020 for $30 million from Radius Health as the race to develop an oral SERD heated up. The FDA also simultaneously approved a companion diagnostic device to identify which patients with breast cancer are suited to take elacestrant. — Max Gelman

  • Company: Eli Lilly
  • Price: $21,000 per 30 days of therapy at the 200 mg dose
  • Peak sales estimate: $1.59 billion – Bloomberg analyst consensus
  • Approval date: 01/27/2023

4. Jaypirca

Active ingredient: pirtobrutinib

Indication: To treat relapsed or refractory mantle cell lymphoma in adults who have had at least two lines of systemic therapy, including a BTK inhibitor + chronic lymphocytic leukemia or small lymphocytic lymphoma (CLL/SLL) who have received at least two prior lines of therapy, including a Bruton’s tyrosine kinase (BTK) inhibitor and a BCL-2 inhibitor

Snapshot: Eli Lilly received two accelerated approvals for its BTK inhibitor Jaypirca to bookend the year. In January, the FDA gave the green light to Jaypirca for mantle cell lymphoma, marking Lilly’s entrance into the BTK field. The approval was for patients whose cancers had returned or stopped responding to treatment, and had at least two prior treatment regimens, including a previous BTK inhibitor. In December, the pill was approved with similar terms for chronic lymphocytic leukemia and small lymphocytic lymphoma. Unlike other approved BTK inhibitors — such as Imbruvica, Calquence and Brukinsa — Lilly’s drug is the first on the market that binds reversibly. The idea is that the non-covalent mechanism can potentially extend the benefit of blocking aberrant B cell receptor signaling that’s seen in these cancers. — Lei Lei Wu

  • Company: GSK
  • Price: Approximately $700 per 30 days of treatment with 6mg tablets for the median patient cohort
  • Peak sales estimate: $269.4 million – Bloomberg analyst consensus
  • Approval date: 02/01/2023

5. Jesduvroq

Active ingredient: daprodustat

Indication: To treat anemia caused by chronic kidney disease for adults on dialysis for at least four months

Snapshot: GSK managed to get Jesduvroq across the finish line after a bumpy development process, but not without the FDA slapping on a safety warning for an increased risk of death, heart attack, stroke and blood clots. Use of the drug was also limited to patients who have been on dialysis for at least four months. Jesduvroq, also known as daprodustat, won approval after the FDA rejected a similar drug from AstraZeneca and FibroGen called roxadustat, citing the need to run another pivotal trial. Getting the drug to patients elsewhere has been a challenge, with GSK announcing plans in July to pull its applications from European and other markets. — Max Gelman

  • Company: Chiesi
  • Price: $4,000 per vial
  • Peak sales estimate: N/A
  • Approval date: 02/16/2023

6. Lamzede

Active ingredient: velmanase alfa-tycv

Indication: To treat non-central nervous system manifestations of alpha-mannosidosis

Snapshot: Chiesi’s velmanase alfa-tycv is an enzyme supplement designed to treat non-central nervous system manifestations of an ultra-rare progressive disease known as alpha-mannosidosis. It occurs when the enzyme α-mannosidase is deficient, resulting in recurrent chest and ear infections, hearing loss, distinctive facial features, muscle weakness and abnormalities in cognition, vision, the skeleton and joints. — Jared Whitlock

  • Company: Travere Therapeutics
  • Price: $9,900 for a 30-day supply; $120,000 per year
  • Peak sales estimate: N/A
  • Approval date: 02/17/2023

7. Filspari

Active ingredient: sparsentan

Indication: To reduce proteinuria in adults with primary immunoglobulin A nephropathy at risk of rapid disease progression

Snapshot: The FDA’s accelerated approval of Travere Therapeutics’ treatment for a rare kidney disease called IgAN was a win for the company in what’s becoming an increasingly crowded field. Travere, which has its roots as one of Martin Shkreli’s old companies, suffered a blow later in the year when a confirmatory trial failed, leading to layoffs and casting doubts on the drug’s future. The company says it will press ahead with a try for full approval, but between the lackluster data and the REMS requirement for the drug, it may be a challenge. — Jared Whitlock

  • Company: Sanofi
  • Price: N/A
  • Peak sales estimate: €2.3 billion – Barclays
  • Approval date: 02/23/2023

8. Altuviiio

Active ingredient: Recombinant Fc-VWF-XTEN fusion protein

Indication: To treat adults and children with hemophilia A (congenital factor VIII deficiency)

Snapshot: Altuviiio added to Sanofi’s portfolio of approved hemophilia A drugs, which already included Eloctate, a treatment that faced an uphill battle after Roche’s Hemlibra came to market. Both of Sanofi’s medicines came via the $11.6 billion acquisition of Bioverativ, and the latest nod for the Sobi-partnered treatment gives the French Big Pharma another chance to compete with Hemlibra. Since the February approval, Altuviiio has already taken some of Hemlibra’s market share. Sanofi could bag another green light for the bleeding disorder this year as the company plans to request approval for the Alnylam-allied fitusiran. — Kyle LaHucik

  • Company: Reata Pharmaceuticals
  • Price: $370,000 per year
  • Peak sales estimate: N/A
  • Approval date: 02/28/2023

9. Skyclarys

Active ingredient: omaveloxolone

Indication: To treat Friedreich’s ataxia

Snapshot: After raising serious efficacy questions that led to a three-month extension of the FDA’s review, the agency in late February ultimately approved Reata Pharmaceuticals’ Skyclarys (omaveloxolone) for a rare genetic disease known as Friedreich’s ataxia, which affects the body’s nerves, for those 16 years and older. The rare disease affects about 6,000 Americans and 22,000 individuals globally. Reata didn’t stay solo for long: Biogen snapped up the biotech in July for $7.3 billion, following a bidding war with another buyer. — Zachary Brennan

  • Company: Pfizer
  • Price: $1,100 per 6 single-dose nasal sprays
  • Peak sales estimate: $1.13 billion – Bloomberg analyst consensus
  • Approval date: 03/09/2023

10. Zavzpret

Active ingredient: zavegepant

Indication: To treat acute migraine with or without aura in adults

Snapshot: In March, Zavzpret became the third drug in Pfizer’s migraine portfolio with an acute indication and the first-and-only CGRP intranasal spray delivery method approved. Zavzpret came to Pfizer in 2022 through the $11.6 billion purchase of Biohaven. The drug’s approval, based on two studies, met co-primary endpoints of pain freedom and freedom from the most bothersome symptoms of migraine two hours after dosage. The intranasal drug adds an alternative for patients with migraine who can’t take pills due to nausea or vomiting. — Beth Bulik

  • Company: Acadia Pharmaceuticals
  • Price: $585,000 on average annually
  • Peak sales estimate: $855 million – Bloomberg analyst consensus
  • Approval date: 03/10/2023

11. Daybue

Active ingredient: trofinetide

Indication: To treat Rett syndrome

Snapshot: Daybue is the first drug for a rare neurodevelopmental disorder called Rett syndrome which is often mistaken for autism or cerebral palsy and primarily affects girls and women. Acadia Pharmaceuticals licensed the synthetic peptide from Neuren Pharmaceuticals in 2018. The drug’s mechanism is unclear, but in 2021, a clinical trial showed that it significantly improved caregiver and physician assessments of a patient’s symptoms. Acadia believes Rett syndrome affects up to 9,000 patients in the US, with half that number currently diagnosed. — Ryan Cross

  • Company: Cidara Therapeutics
  • Price: $1,950 per vial (200mg) 
  • Peak sales estimate: N/A
  • Approval date: 03/22/2023

12. Rezzayo

Active ingredient: rezafungin

Indication: To treat candidemia and invasive candidiasis

Snapshot: It’s been more than a decade since there was a new treatment for the fungal infections candidemia and invasive candidiasis, according to Melinta Therapeutics and Cidara Therapeutics. The drug was approved after showing statistical non-inferiority to caspofungin, a drug by Merck approved in 2011 to treat Candida infections and which serves as the current standard. Rezzayo was developed by Cidara, which still has rights in Japan, while Melinta owns the US rights. Sackler family-owned British multinational Mundipharma has rights in all other geographies. — Ngai Yeung

  • Company: Incyte
  • Price: $14,240 per vial
  • Peak sales estimate: $361.6 million – Bloomberg analyst consensus
  • Approval date: 03/22/2023

13. Zynyz

Active ingredient: retifanlimab-dlwr

Indication: To treat metastatic or recurrent locally advanced Merkel cell carcinoma

Snapshot: After the FDA rejected it for a type of anal cancer, Incyte’s PD-1 inhibitor Zynyz won an accelerated approval from the agency in March to treat metastatic or recurrent locally advanced Merkel cell carcinoma, a rare and fast-growing skin cancer. The approval was based on positive results from the company’s single-arm POD1UM-201 study, which is ongoing and will serve as the confirmatory trial. Zynyz is the eighth PD-1/PD-L1 inhibitor to win FDA approval. — Lia DeGroot

  • Company: Pharming Group
  • Price: $45,000 for a 30-day supply
  • Peak sales estimate: N/A
  • Approval date: 03/24/2023

14. Joenja

Active ingredient: leniolisib

Indication: To treat activated phosphoinositide 3-kinase delta syndrome

Snapshot: Joenja scored approval from the FDA in March as the first and only approved treatment for activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome, which triggers immunodeficiency in patients. The drug belongs to Pharming Group and marks the company’s second commercial product. Joenja is still under consideration from the EMA, which is predicted to make a decision in the first quarter of 2024. — Anna Brown

  • Company: Biogen
  • Price: $14,230 per dose
  • Peak sales estimate: $242.1 million – Bloomberg analyst consensus
  • Approval date: 04/25/2023

15. Qalsody

Active ingredient: tofersen

Indication: To treat amyotrophic lateral sclerosis in adults who have a SOD1 gene mutation

Snapshot: The FDA’s approval of Qalsody was only for a tiny fraction of amyotrophic lateral sclerosis (ALS) patients with a SOD1 mutation — about 300 people in the US — but the decision may influence future treatment options for many more people with ALS and other neurodegenerative diseases. After the drug failed to hit its primary endpoint in a Phase III study, the FDA granted accelerated approval to Qalsody based on longer-term follow-up results, as well as the drug’s ability to reduce neurofilament, a protein shed by damaged neurons that is tied to many neurodegenerative conditions. In March, an FDA adcomm voted unanimously that Qalsody’s impact on neurofilament could predict clinical benefit, and the FDA approval reiterated that the protein could be a surrogate biomarker. — Lei Lei Wu

  • Company: Seres Therapeutics
  • Price: $17,500 per course (three days)
  • Peak sales estimate: $850 million – TD Cowen
  • Approval date: 04/26/2023

16. Vowst

Active ingredient: fecal microbiota spores, live-brpk

Indication: To prevent recurrence of Clostridioides difficile (C. difficile) infection (CDI) in individuals 18 years of age and older

Snapshot: Seres snagged its first approval, and it marked a big moment for poop. The Cambridge, MA-based biotech, with the help of Nestlé Health Science, landed a key moment for the microbiome field when the FDA cleared the duo’s pill that is purified from human stool. The move came a few months after Ferring Pharmaceuticals also gained a green light to market a C. difficile medicine, known as Rebyota. — Kyle LaHucik

  • Company: GSK
  • Price: $280 per dose
  • Peak sales estimate: GSK expects peak sales greater than £3 billion
  • Approval date: 05/03/2023

17. Arexvy

Active ingredient: RSVPreF3

Indication: To prevent lower respiratory tract disease caused by RSV in adults 60 years and older

Snapshot: Arexvy was the first approved RSV vaccine in the US. GSK’s chief commercial officer Luke Miels said at a November investor conference that Arexvy has taken over 71% of what’s expected to be a multibillion-dollar market for RSV preventatives. It’s in direct competition with Pfizer’s Abrysvo, which was approved in May. — Nicole DeFeudis

  • Company: Chiesi Global Rare Diseases and Protalix BioTherapeutics
  • Price: $4,135.11 for a 20-milligram vial; yearly price of about $350,000 to $400,000
  • Peak sales estimate: N/A
  • Approval date: 05/09/2023

18. Elfabrio

Active ingredient: pegunigalsidase alfa-iwxj

Indication: To treat confirmed Fabry disease

Snapshot: Enzyme replacement therapy Elfabrio is the second approved drug using Protalix’s platform, which produces recombinant proteins with plant cells. The Israel-based biotech partnered with Chiesi to sell Elfabrio. The two companies originally applied for an accelerated approval in 2021, which the agency rejected. The rare kidney disease now has a handful of treatment options, including Elfabrio, Amicus Therapeutics’ Galafold, and Sanofi’s Fabrazyme. — Andy Dunn

  • Company: Astellas
  • Price: $550 for a 30-day supply
  • Peak sales estimate: $1.46 billion – Bloomberg analyst consensus
  • Approval date: 05/12/2023

19. Veozah

Active ingredient: fezolinetant

Indication: To treat moderate to severe hot flashes caused by menopause

Snapshot: Astellas originally expected a decision on its nonhormonal hot flash treatment Veozah in February, having spent a priority review voucher to speed up the process by four months. But the FDA took extra time to review the package and didn’t approve it until May, based on three Phase III trials. The company priced the drug considerably higher than ICER’s previously recommended range of $2,000 to $2,500 a year. — Ngai Yeung

  • Company: Bausch + Lomb and Novaliq
  • Price: $771 for a one-month supply (3 mL of Miebo in a 5mL bottle, or approximately 270 drops)
  • Peak sales estimate: N/A
  • Approval date: 05/18/2023

20. Miebo

Active ingredient: perfluorohexyloctane

Indication: To treat signs and symptoms of dry eye disease

Snapshot: Miebo is eye health giant Bausch + Lomb’s first FDA-approved prescription eye treatment since the company went public last year. The drug, approved in May based on two Phase III studies, is designed to target tear evaporation, a leading cause of dry eye disease. The disease affects millions of Americans and is one of the most common eye surface disorders, the company said. — Ngai Yeung

  • Company: AbbVie and Genmab
  • Price: $37,500 per month
  • Peak sales estimate: N/A
  • Approval date: 05/19/2023

21. Epkinly

Active ingredient: epcoritamab-bysp

Indication: To treat relapsed or refractory diffuse large B-cell lymphoma (not otherwise specified) and high-grade B-cell lymphoma after two or more lines of systemic therapy

Snapshot: AbbVie and Genmab came in second to Roche and Genentech’s Lunsumio when they got an Epkinly approval in May, but the CD3xCD20 drug is the first subcutaneous bispecific antibody approved as a third-line treatment for relapsed/refractory large B cell lymphoma. The approval put a bow on a 2020 alliance between the companies when AbbVie paid $750 million upfront to partner with Genmab and promised as much as $3.15 billion in milestones. The pair plans to keep testing the drug for other blood cancers. Epkinly was one of four bispecific T cell engagers approved by the FDA in 2023 in large B cell lymphoma or multiple myeloma, in addition to Columvi, Elrexfio and Talvey. — Max Gelman

  • Company: Krystal Biotech
  • Price: $24,250 per vial, with the average patient using 26 vials per year; Annual cost of about $631,000 per patient
  • Peak sales estimate: N/A
  • Approval date: 05/19/2023

22. Vyjuvek

Active ingredient: beremagene geperpavec-svdt

Indication: To treat wounds in patients 6 months of age and older with dystrophic epidermolysis bullosa with mutation(s) in the collagen type VII alpha 1 chain (COL7A1) gene

Snapshot: Vyjuvek in May became the first approved treatment in the US for “butterfly children” — kids born with a rare genetic condition called dystrophic epidermolysis bullosa that makes their skin incredibly fragile. The gel treatment, marketed by Krystal Biotech, is applied to wounded skin, and unlike other gene therapies which can only be dosed once, the herpes virus-based gene therapy gel can be re-applied. Krystal Biotech is now investigating an eye drop version of the treatment that could potentially help patients with vision complications or blindness from the genetic condition. — Lei Lei Wu

  • Company: Entasis Therapeutics
  • Price: $1,900 per day; average course of treatment for patients in PhIII was 7-10 days
  • Peak sales estimate: ~$110 million – Goldman Sachs
  • Approval date: 05/23/2023

23. Xacduro

Active ingredient: sulbactam, durlobactam

Indication: To treat hospital-acquired bacterial pneumonia and ventilator-associated bacterial pneumonia caused by susceptible isolates of Acinetobacter baumannii-calcoaceticus complex

Snapshot: In May, the FDA approved Xacduro for certain pneumonia infections that are most frequently seen in hospitals. The dual antibiotic targets a type of bacteria called Acinetobacter baumannii-calcoaceticus complex, which predominantly causes pneumonia and can be highly resistant to current drugs. Innoviva subsidiary Entasis Therapeutics pushed the antibiotic forward after AstraZeneca cut the program as part of a larger retreat from antibiotics research. The FDA highlighted the importance of the approval for these difficult-to-treat infections — but whether Xacduro can succeed commercially in a market that makes it difficult for new antibiotics to turn a profit remains to be seen. — Lei Lei Wu

  • Company: Blue Earth Diagnostics
  • Price: $4,775
  • Peak sales estimate: N/A
  • Approval date: 05/25/2023

24. Posluma

Active ingredient: flotufolastat F 18

Indication: To use with positron emission tomography (PET) imaging in certain patients with prostate cancer

Snapshot: The FDA approved Posluma, a positron emission tomography imaging agent, for individuals with metastasized prostate cancer who are candidates for therapy or with suspected recurrence based on elevated PSA levels. — Jared Whitlock

  • Company: Pfizer
  • Price: $1,390 for a five-day course
  • Peak sales estimate: $18.9 billion in 2022
  • Approval date: 05/25/2023

25. Paxlovid

Active ingredient: nirmatrelvir, ritonavir

Indication: To treat mild-to-moderate Covid-19 in adults at high risk for progression to severe Covid-19

Snapshot: Covid-19 pandemic stalwart Paxlovid needs little introduction. The protease inhibitor won a full FDA approval in May to treat mild-to-moderate Covid-19 in high-risk adults after being on the market since December 2021 under an emergency use authorization. It raked in $19 billion in 2022 sales, likely making it the lone case of a mega-blockbuster that peaked before earning an official FDA approval. Pfizer has since seen 2023 sales come in below initial expectations, and the pharma giant is preparing to transition from selling to the US government to the commercial market in 2024. — Andy Dunn

  • Company: Lexicon Pharmaceuticals
  • Price: $598 per month
  • Peak sales estimate: Consensus sees peak sales of $697 million in 2031; Jefferies sees peak sales reaching $332 million
  • Approval date: 05/26/2023

26. Inpefa

Active ingredient: sotagliflozin

Indication: To treat heart failure and related hospitalizations, including type 2 diabetes and chronic kidney disease

Snapshot: The FDA approved Lexicon’s heart failure drug Inpefa in May. The sodium-glucose cotransporter 2 (SGLT2) inhibitor is used to reduce the risk of cardiovascular death, as well as heart failure-related hospitalization in adults with cardiovascular-related diseases, such as type 2 diabetes. Lexicon only won approval after a series of hiccups, including a 2019 FDA rejection of Inpefa as a treatment for diabetes, which led partner Sanofi to cancel its $1.7 billion deal with the company a few months later. — Anna Brown

  • Company: Pfizer
  • Price: $295 per dose
  • Peak sales estimate: N/A
  • Approval date: 05/31/2023

27. Abrysvo

Active ingredient: RSVpreF

Indication: To prevent lower respiratory tract disease caused by RSV in adults 60 years and older

Snapshot: Abrysvo was approved in May for the prevention of RSV in older adults, then again in August to help protect newborns when administered to women between 32 and 36 weeks of pregnancy. It’s the first maternal vaccine to help protect newborns against lower respiratory tract disease from RSV through 6 months of age. —Nicole DeFeudis

  • Company: Roche
  • Price: $350,000 for an 8.5 month course
  • Peak sales estimate: $1.23 billion – Bloomberg analyst consensus
  • Approval date: 06/15/2023

28. Columvi

Active ingredient: glofitamab-gxbm

Indication: To treat diffuse large B-cell lymphoma, not otherwise specified, or large B-cell lymphoma arising from follicular lymphoma after two or more lines of systemic therapy

Snapshot: Roche expanded its bispecific antibody franchise in June with an accelerated approval of Columvi as a third-line treatment for an aggressive form of non-Hodgkin’s lymphoma. It’s the company’s second CD20xCD3 therapy designed to connect T cells to blood cancers, equipped with two antibody arms targeting the blood cell marker CD20 to make it more potent than its predecessor, Lunsumio. In a Phase I/II trial, 56% of patients responded to an 8.5-month course of Columvi, and 43% had a complete response. — Ryan Cross

  • Company: Sarepta Therapeutics
  • Price: $3.2 million
  • Peak sales estimate: N/A
  • Approval date: 06/22/2023

29. Elevidys

Active ingredient: delandistrogene moxeparvovec-rokl

Indication: To treat ambulatory pediatric patients aged 4 through 5 years with Duchenne muscular dystrophy (DMD) with a confirmed mutation in the DMD gene

Snapshot: The FDA’s accelerated approval of Sarepta’s Duchenne muscular dystrophy gene therapy was one of the most closely-watched regulatory decisions of 2023. Elevidys was approved for a narrow group of boys with Duchenne — those ages 4 through 5 — after senior FDA official Peter Marks overrode reviewers’ calls to reject the therapy. In October, the gene therapy’s confirmatory study read out with mixed results, but analysts have said that it’s unlikely the FDA will pull the one-time treatment from the market based on the data. — Lei Lei Wu

  • Company: Pfizer
  • Price: $49,000 per year
  • Peak sales estimate: $914.6 million – Bloomberg analyst consensus
  • Approval date: 06/23/2023

30. Litfulo

Active ingredient: ritlecitinib

Indication: To treat severely patchy hair loss

Snapshot: Pfizer may have followed behind Eli Lilly’s Olumiant in getting its drug Litfulo approved for alopecia, but the New York drug giant won FDA clearance for both adults and teens, giving it access to a broader patient population. Litfulo comes with a boxed safety warning, like other JAK inhibitors. In Litfulo’s pivotal trial, 23% of patients on the 50 mg dose had 80% or more scalp hair coverage after six months, compared to just 2% in the placebo group. Litfulo is also being studied to treat vitiligo, ulcerative colitis and Crohn’s disease. — Max Gelman

  • Company: UCB
  • Price: $6,050 per vial
  • Peak sales estimate: $800 million – Bloomberg analyst consensus
  • Approval date: 06/26/2023

31. Rystiggo

Active ingredient: rozanolixizumab-noli

Indication: To treat generalized myasthenia gravis in adults who are anti-acetylcholine receptor- or anti-muscle-specific tyrosine kinase antibody-positive

Snapshot: The FDA in June approved UCB’s Rystiggo to treat generalized myasthenia gravis, a rare autoimmune disease. The Belgian company said it’s the first option to treat the two main subtypes of the disease. The approval, which was granted priority review, came on the heels of argenx’s myasthenia gravis drug Vyvgart Hytrulo. Rystiggo works by targeting the neonatal Fc receptor in hopes of reducing levels of immunoglobulin G antibodies, which impede the messaging between nerves and muscles. — Lia DeGroot

  • Company: Pfizer and OPKO Health
  • Price: $8,300 monthly list price, assuming a 35 kg child
  • Peak sales estimate: $350 million – Bloomberg analyst consensus
  • Approval date: 06/27/2023

32. Ngenla

Active ingredient: somatrogon-ghla

Indication: To treat growth failure due to inadequate secretion of endogenous growth hormone

Snapshot: Pfizer and the Miami-based healthcare company OPKO Health started working together in 2014 on a longer-acting treatment for growth hormone deficiency. Nearly a decade later, and after an initial rejection in 2022, the FDA approved Ngenla in June for children as young as three years old. Pfizer is in charge of selling the drug, which will compete against the Danish drugmaker Ascendis Pharma’s Skytrofa, a weekly injection like Pfizer’s drug. — Andy Dunn

  • Company: CellTrans
  • Price: N/A
  • Peak sales estimate: N/A
  • Approval date: 06/28/2023

33. Lantidra

Active ingredient: donislecel

Indication: To treat type 1 diabetes

Snapshot: Lantidra is the first cell therapy approved to treat type 1 diabetes. The treatment, which involves taking pancreatic cells from deceased donors, is available to patients who are unable to approach average blood glucose levels because of low blood sugar even after intensive management. It’s administered as a single infusion, and the goal is that patients will no longer need insulin. — Nicole DeFeudis

  • Company: BioMarin
  • Price: $2.9 million
  • Peak sales estimate:$2.2 billion, per Biopharma Dive
  • Approval date: 06/29/2023

34. Roctavian

Active ingredient: valoctocogene roxaparvovec-rvox

Indication: To treat adults with severe hemophilia A without antibodies to adeno-associated virus serotype 5 detected by an FDA-approved test

Snapshot: Roctavian is the first gene therapy approved to treat hemophilia A, a genetic bleeding disorder. It was approved in June following a 2020 rejection and a review extension in 2023, and BioMarin hopes to turn it into a key blockbuster drug. — Nicole DeFeudis

  • Company: Sanofi and AstraZeneca
  • Price: Commercial: $495; Vaccines for children program: $395
  • Peak sales estimate: $3 billion – Jefferies 
  • Approval date: 07/17/2023

35. Beyfortus

Active ingredient: nirsevimab-alip

Indication: To prevent respiratory syncytial virus (RSV)

Snapshot: In July, the FDA approved Beyfortus, an antibody drug co-developed by Sanofi and AstraZeneca. Beyfortus, also known as nirsevimab, is given as a one-time intramuscular injection to infants, protecting them from respiratory syncytial virus, or RSV. Beyfortus is the latest in a suite of new medicines against RSV, a virus that typically puts 58,000 to 80,000 young children in the US in the hospital every year, according to the CDC. — Jared Whitlock

  • Company: Emergent BioSolutions
  • Price: N/A
  • Peak sales estimate: N/A
  • Approval date: 07/20/2023

36. Cyfendus

Active ingredient: anthrax vaccine adsorbed, adjuvanted

Indication: Post-exposure prophylaxis after exposure to Bacillus anthracis

Snapshot: Cyfendus is used for post-exposure prophylaxis after suspected or confirmed exposure to the bacteria that causes anthrax, Bacillus anthracis. While the two-dose vaccine received full approval in 2023, it has been provided to the US government for years under pre-emergency use authorization. — Nicole DeFeudis

  • Company: Daiichi Sankyo
  • Price: $546 per tablet for 7.7 mg and 26.5 mg doses
  • Peak sales estimate: N/A
  • Approval date: 07/20/2023

37. Vanflyta

Active ingredient: quizartinib

Indication: To use as part of a treatment regimen for newly diagnosed acute myeloid leukemia that meets certain criteria

Snapshot: Daiichi Sankyo won FDA approval for Vanflyta in July as a frontline treatment for acute myeloid leukemia. This came after the agency delayed a decision on the approval in April — extending the PDUFA date by three months to review more data from the company. As well as being used as a first-line treatment in combination with chemo, Vanflyta was also approved as a maintenance monotherapy. — Anna Brown

  • Company: Octapharma
  • Price: N/A
  • Peak sales estimate: N/A
  • Approval date: 07/21/2023

38. Balfaxar

Active ingredient: prothrombin complex concentrate, human-lans

Indication: To help restore blood coagulation in patients on blood thinners before urgent surgeries or invasive procedures

Snapshot: Balfaxar is approved for adults who need an urgent reversal of blood thinners such as warfarin ahead of surgeries or other invasive procedures. While blood thinners are prescribed to more than 2.4 million Americans to reduce the risk of blood clots, they also put those patients at a higher risk of heavy bleeding in surgery. — Nicole DeFeudis

  • Company: Tarsus Pharmaceuticals
  • Price: $1,850 for six-week treatment course
  • Peak sales estimate: Peak sales in the US approaching $1 billion by 2030 – William Blair
  • Approval date: 07/25/2023

39. Xdemvy

Active ingredient: lotilaner

Indication: To treat Demodex blepharitis

Snapshot: Tarsus’ first-ever drug approval, Xdemvy, came earlier than expected in July, a month before its FDA deadline. It’s the first-ever drug approved to treat Demodex blepharitis, eye irritation and inflammation caused by tiny mites that are often mistaken for allergy symptoms. The eye drops are a reformulation of a veterinary drug used to prevent fleas and ticks, and Tarsus’ studies showed significant improvement in both mite eradication (52% of treated patients) and reduction of yellowish eyelid collarettes (89% of treated patients) at six weeks. — Beth Bulik

  • Company: Iveric Bio
  • Price: $2,100 per single-dose vial
  • Peak sales estimate: FY2025 – peak sales of ¥200 billion to ¥400 billion (~1.4 billion – 2.8 billion USD) -Astellas; Global peak sales ¥500 billion – Jefferies
  • Approval date: 08/04/2023

40. Izervay

Active ingredient: avacincaptad pegol

Indication: To treat geographic atrophy secondary to age-related macular degeneration

Snapshot: A few months after being bought by Japanese pharma company Astellas for $5.9 billion, Iveric Bio secured an FDA green light for its geographic atrophy treatment Izervay. Competitor Apellis Pharmaceuticals had snagged the first-ever approval for the condition, which is a leading cause of blindness, earlier in the year. The back-to-back nods set up a showdown over safety and efficacy. Izervay’s use was capped at 12 months, whereas Apellis’ Syfovre has no limitations, but the biotech presented more data in September hoping to expand its label. — Kyle LaHucik

  • Company: Biogen and Sage Therapeutics
  • Price: $15,900 for a one-time two-week course
  • Peak sales estimate: $655.7 million – Bloomberg analyst consensus
  • Approval date: 08/04/2023

41. Zurzuvae

Active ingredient: zuranolone

Indication: To treat postpartum depression

Snapshot: After decades without any pharmaceutical progress, mothers suffering from postpartum depression finally have a powerful option. The fast-acting drug Zurzuvae can dramatically lift moods in a matter of days. It’s a highly awaited sequel to Sage’s onerous 60-hour infusion for the condition that was a commercial failure. Zurzuvae was inspired by a natural hormone whose falling levels may trigger postpartum depression and target GABA receptors in the brain. But Zurzuvae’s effectiveness was murky in major depressive disorder, a far larger market, leading the FDA to reject that broader use and leaving the drug’s and Sage’s future uncertain. — Ryan Cross

  • Company: Johnson & Johnson
  • Price: $45,000 per month
  • Peak sales estimate: $2.38 billion – Bloomberg analyst consensus
  • Approval date: 08/09/2023

42. Talvey

Active ingredient: talquetamab-tgvs

Indication: To treat adults with relapsed or refractory multiple myeloma who have received at least four prior therapies

Snapshot: Johnson & Johnson is on a roll in blood cancer. Talvey’s accelerated approval gives the company five marketed multiple myeloma drugs, and it joins a growing arsenal of medicines for multiple myeloma, including J&J and Legend Biotech’s CAR-T Carvykti, Bristol Myers Squibb’s Abecma, Pfizer’s newly cleared Elrexfio and others. The bispecific antibody, injected under the skin weekly or biweekly, goes after the CD3 receptor on the T cell’s surface and GPRC5D on multiple myeloma cells, among other cells. It comes with a boxed warning for cytokine release syndrome. — Kyle LaHucik

  • Company: Pfizer
  • Price: $41,500 per month
  • Peak sales estimate: $733.2 million – Bloomberg analyst consensus
  • Approval date: 08/14/2023

43. Elrexfio

Active ingredient: elranatamab-bcmm

Indication: To treat adults with relapsed or refractory multiple myeloma who have received at least four prior lines of therapy

Snapshot: Pfizer expects that Elrexfio could become a “multibillion-dollar franchise” following its accelerated approval in August for adults with multiple myeloma. The subcutaneous injection was cleared for relapsed or refractory multiple myeloma patients who have previously received at least four prior therapies, including a proteasome inhibitor, an immunomodulatory agent and an anti-CD38 monoclonal antibody. It works by targeting BCMA on myeloma cells and CD3 on T cells, which prompts the T cells to attack the myeloma cells.  — Lia DeGroot

  • Company: Ipsen
  • Price: Estimated annual list price of $624,000
  • Peak sales estimate: Over €100 million – Ipsen
  • Approval date: 08/16/2023

44. Sohonos

Active ingredient: palovarotene

Indication: To reduce the volume of new heterotopic ossification in adults and pediatric patients (aged 8 years and older for females and 10 years and older for males) with fibrodysplasia ossificans progressiva

Snapshot: Ipsen’s Sohonos was approved for the treatment of the ultra-rare disease fibrodysplasia ossificans progressiva despite the FDA’s questions over its efficacy and safety. The agency’s OK came after a 2022 rejection, in which regulators asked for more data from existing studies. Ipsen resubmitted its application and said Sohonos should come with a boxed warning about premature growth plate closure. The drug also eventually got a 10-4 vote in favor of its benefit-risk profile from the FDA’s Endocrinologic and Metabolic Drugs Advisory Committee, and approval followed. — Katherine Lewin

  • Company: Regeneron
  • Price: $34,600 per single-use vial
  • Peak sales estimate: N/A
  • Approval date: 08/18/2023

45. Veopoz

Active ingredient: pozelimab-bbfg

Indication: To treat patients 1 year and older with CD55-deficient protein-losing enteropathy (PLE), also known as CHAPLE disease

Snapshot: Veopoz is approved to treat CHAPLE disease, a rare but potentially life-threatening immune disease that affects fewer than 100 people worldwide. It targets a protein called complement factor C5, which is involved in the activation of a part of the immune system called the complement system. Beyond CHAPLE disease, executives have said the drug has promise in a variety of other complement-mediated diseases. — Nicole DeFeudis

  • Company: BioLineRx
  • Price: $5,900 per vial
  • Peak sales estimate: N/A
  • Approval date: 09/08/2023

46. Aphexda

Active ingredient: motixafortide

Indication: To use with filgrastim (G-CSF) to mobilize hematopoietic stem cells to the peripheral blood for collection and subsequent autologous transplantation in patients with multiple myeloma

Snapshot: Autologous stem cell transplantation is part of the standard of care for multiple myeloma patients, a process in which a patient’s stem cells are removed and reintroduced after treatment with chemotherapy. But with some patients, there’s trouble collecting enough hematopoietic stem cells in one round, leading to sessions that can last hours a day for up to five days. BioLineRx’s drug, Aphexda, is used in combination with filgrastim to make the cells easier to collect. — Nicole DeFeudis

  • Company: GSK
  • Price: $26,900 for a 30-tablet bottle
  • Peak sales estimate: $861 million – Bloomberg analyst consensus
  • Approval date: 09/15/2023

47. Ojjaara

Active ingredient: momelotinib

Indication: To treat intermediate or high-risk myelofibrosis in adults with anemia

Snapshot: Ojjaara’s approval in September for myelofibrosis patients with anemia capped off a long turnaround story for the JAK inhibitor. The drug was shelved by Gilead after a failed study and then licensed by Sierra Oncology for only $3 million upfront in 2018. After Sierra successfully steered the candidate through a new late-stage study, GSK swooped in with $1.9 billion to buy the company. Ojjaara was the fourth JAK inhibitor approved for the rare bone marrow cancer, but clinical studies suggest that, unlike earlier JAK inhibitors, it can help control anemia instead of causing or worsening it — a boon for a patient population in which many develop anemia. — Lei Lei Wu

  • Company: Fabre-Kramer Pharmaceuticals
  • Price: N/A
  • Peak sales estimate: N/A
  • Approval date: 09/22/2023

48. Exxua

Active ingredient: gepirone

Indication: To treat major depressive disorder

Snapshot: The biotech industry got a surprise in late September when a largely forgotten company said it had received FDA approval for its major depressive disorder tablet Exxua. The move came about three months later than expected because of “major amendments” to the filing that needed to be addressed, but the approval was actually two decades later than the drug’s developers had initially liked. Houston-based Fabre-Kramer Pharmaceuticals and its partners received multiple no-gos from the FDA and its outside advisors dating all the way back to 2002. The drug shuffled through the hands of Organon, GSK and back to Fabre-Kramer during its multi-decade journey. — Kyle LaHucik

  • Company: Amicus Therapeutics
  • Price: $650,000 for Pombiliti and Opfolda for a patient weighing approximately 70 kg
  • Peak sales estimate: $706.8 million – Bloomberg analyst consensus
  • Approval date: 09/28/2023

49. Pombiliti

Active ingredient: cipaglucosidase alfa-atga

Indication: To treat late-onset Pompe disease

Snapshot: The FDA approved Pombiliti, a long-term enzyme replacement therapy, and Opfolda, an enzyme stabilizer, as a combo therapy for Pompe disease. Amicus Therapeutics hit several regulatory delays along the way to the September approval — Phase III data for the therapy showed it did not beat out Sanofi’s drug Lumizyme. — Katherine Lewin

  • Company: Novo Nordisk
  • Price: Rivfloza will be priced “to reflect its demonstrated clinical value and benefit to those living with PH1,” a spokesperson said.
  • Peak sales estimate: N/A
  • Approval date: 09/29/2023

50. Rivfloza

Active ingredient: nedosiran

Indication: To lower urinary oxalate levels in patients 9 years and older with primary hyperoxaluria type 1 and relatively preserved kidney function

Snapshot: Rivfloza is an RNAi therapy developed by Dicerna to treat a rare kidney disease that’s marked by an excessive production of oxalate, a substance that can combine with calcium to cause kidney stones. The drug lowers urinary oxalate levels, and will be available in early 2024, according to Novo Nordisk. It will compete with Alnylam’s rival Oxlumo, which was approved in 2020. — Nicole DeFeudis

  • Company: Pfizer
  • Price: $6,164 for a 30-day bottle
  • Peak sales estimate: $2.02 billion – Bloomberg analyst consensus
  • Approval date: 10/12/2023

51. Velsipity

Active ingredient: etrasimod

Indication: To treat moderate to severe plaque psoriasis in adults who are candidates for systemic therapy or phototherapy

Snapshot: Velsipity was at the center of Pfizer’s $6.7 billion acquisition of Arena Pharmaceuticals. It’s the second selective sphingosine-1-phosphate (S1P) receptor modulator approved to treat ulcerative colitis behind Bristol Myers Squibb’s Zeposia, though it will also contend with a variety of other treatments from steroids to biologics. Velsipity and Zeposia are contraindicated in patients with certain heart issues and carry warnings for bradyarrhythmia, where the heart rate temporarily slows down. — Nicole DeFeudis

  • Company: UCB
  • Price: $7,200 per syringe
  • Peak sales estimate: $3.43 billion – Bloomberg analyst consensus
  • Approval date: 10/17/2023

52. Bimzelx

Active ingredient: bimekizumab

Indication: To treat moderate to severe plaque psoriasis in adults who are candidates for systemic therapy or phototherapy

Snapshot: UCB’s Bimzelx overcame headwinds at the company’s Belgian manufacturing plant to win US approval in plaque psoriasis in October, two years after a thumbs-up in Europe. The IL-17A/IL-17F inhibitor was rejected by the FDA in early 2022 and faced further delays in its review process later that year. After satisfying the FDA that the manufacturing issues were resolved, Bimzelx was launched in the US in November. The Belgian drugmaker believes the product can reach sales of at least €4 billion ($4.23 billion), despite warnings on its label for liver monitoring and suicidal ideation. — Ayisha Sharma

  • Company: UCB
  • Price:
  • Peak sales estimate: $528 million – TD Cowen
  • Approval date: 10/17/2023

53. Zilbrysq

Active ingredient: zilucoplan

Indication: To treat generalized myasthenia gravis in adults who are anti-acetylcholine receptor (AChR) antibody-positive

Snapshot: UCB’s peptide approach to complement C5 inhibition got the go-ahead from the FDA in October for generalized myasthenia gravis patients who are anti-acetylcholine receptor (AChR) antibody-positive. Unlike traditional C5-targeting monoclonal antibodies, Zilbrysq can be used alongside intravenous immunoglobulin and plasma exchange without supplemental dosing. According to TD Cowen analysts, Zilbrysq’s once-daily subcutaneous dosing regimen lines up with patient wants, who projected at least €500 million ($528 million) in sales. — Ayisha Sharma

  • Company: Eli Lilly
  • Price: $9,593 per month for initial IV infusion then $10,360 per under-the-skin injection thereafter
  • Peak sales estimate: $1.51 billion – Bloomberg analyst consensus
  • Approval date: 10/26/2023

54. Omvoh

Active ingredient: mirikizumab-mrkz

Indication: To treat ulcerative colitis

Snapshot: Lilly entered the big inflammatory bowel disease market thanks to a second chance with Omvoh. The pharma giant had ditched plans to request approval in psoriasis in 2021, then moved forward in ulcerative colitis but received an FDA rejection in April because of manufacturing issues. Those issues were resolved and the FDA gave it a nod in October. The Indianapolis drugmaker had recently beefed up its autoimmune pipeline via the $2.4 billion purchase of DICE Therapeutics. In a similar area, Lilly seeks to mark a turnaround for its eczema drug lebrikizumab, also rejected in 2023 due to manufacturing issues. — Kyle LaHucik

  • Company: Santhera
  • Price: N/A
  • Peak sales estimate: $500 million – 2021 estimate from Santhera
  • Approval date: 10/26/2023

55. Agamree

Active ingredient: vamorolone

Indication: To treat Duchenne muscular dystrophy

Snapshot: After multiple setbacks, Santhera’s commitment to Duchenne muscular dystrophy finally paid off when its steroid-based drug Agamree won FDA approval in October. Agamree was meant to be filed in 2022, but third-party manufacturing issues delayed the process by several months. The approval gave a much-needed win, after Santhera’s previous Duchenne candidate, idebenone, failed a Phase III trial in 2020 and was discontinued. Agamree is designed to address the problem of dose-limiting toxicities linked with more traditional steroids. — Ayisha Sharma

  • Company: Coherus
  • Price: $8,892.03 per single-use vial
  • Peak sales estimate: $200 million peak in US – Coherus; 2030 sales of $160 million – TD Cowen 
  • Approval date: 10/27/2023

56. Loqtorzi

Active ingredient: toripalimab-tpzi

Indication: To treat recurrent or metastatic nasopharyngeal carcinoma when used together with or following other therapies

Snapshot: Junshi Biosciences made history in China by bringing the first homegrown PD-1 to the market five years ago. Together with partner Coherus, it also beat much bigger players in ushering the first Chinese checkpoint inhibitor into the US, winning approval for Loqtorzi in patients with recurrent or metastatic nasopharyngeal carcinoma across all lines of treatment. The approval followed an initial rejection and subsequent review delay. Going after a cancer type that’s prevalent in southeast Asia but relatively rare in the US, Junshi and Coherus won over the FDA with clinical data from just China and several other Asian countries — something that the agency deemed insufficient in bigger indications. — Amber Tong

  • Company: Takeda
  • Price: N/A
  • Peak sales estimate: $144.9 million – Bloomberg analyst consensus
  • Approval date: 11/08/2023

57. Fruzaqla

Active ingredient: fruquintinib

Indication: To treat refractory, metastatic colorectal cancer

Snapshot: The FDA’s approval for Fruzaqla may have gone to Takeda, but it’s also a momentous decision for Hutchmed, which first won approval for the VEGF receptor kinase inhibitor in China in 2018 under a different name. Takeda bought the ex-China global rights for $400 million upfront at the beginning of the year. In clinical trials, the oral drug was shown to improve both overall survival and progression-free survival in patients with previously treated metastatic colorectal cancer, a space that Takeda says has seen few new treatments over the years. — Amber Tong

  • Company: CorMedix
  • Price: N/A
  • Peak sales estimate: N/A
  • Approval date: 11/15/2023

58. Defencath

Active ingredient: taurolidine, heparin

Indication: To reduce the incidence of catheter-related bloodstream infections in adults with kidney failure receiving chronic hemodialysis through a central venous catheter

Snapshot: Defencath is approved to reduce catheter-related bloodstream infections in adults with kidney failure. Its use is limited to patients who are receiving chronic hemodialysis through a central venous catheter. It’s the third drug to be approved via the FDA’s Limited Population Pathway for Antibacterial and Antifungal Drugs (LPAD) from 2016. CorMedix previously received two complete response letters for Defencath, both of which cited contract manufacturing issues. — Nicole DeFeudis

  • Company: Bristol Myers Squibb
  • Price: $29,000 per month for adult patients
  • Peak sales estimate: $997.4 million – Bloomberg analyst consensus
  • Approval date: 11/15/2023

59. Augtyro

Active ingredient: repotrectinib

Indication: To treat ROS1-positive non-small cell lung cancer

Snapshot: Augtyro scored approval in November, adding another treatment option for adult patients with locally advanced or metastatic ROS1-positive non-small cell lung cancer. It’s a rival to Pfizer’s Xalkori and Roche’s Rozlytrek in ROS1-positive NSCLC, and marked a payoff for Bristol Myers Squibb after it acquired the drug when it bought Turning Point Therapeutics for $4.1 billion in June 2022. — Katherine Lewin

  • Company: AstraZeneca
  • Price:$22,922 for a 28-day supply of the medicine
  • Peak sales estimate: $1.44 billion – Bloomberg analyst consensus
  • Approval date: 11/16/2023

60. Truqap

Active ingredient: capivasertib

Indication: To treat breast cancer that meets certain disease criteria

Snapshot: With the approval of Truqap, AstraZeneca expanded its breast cancer portfolio to offer patients who have hormone receptor-positive, HER2-negative tumors another targeted therapy option. However, the label comes with a restriction: The AKT inhibitor is approved in combination with AstraZeneca’s own Faslodex to treat a subset of patients whose tumors harbor certain genetic alterations, a narrower population than some analysts expected based on late-stage data showing progression-free survival improvement for all-comers. Testing is required to prescribe the drug. AstraZeneca first discovered Truqap via a longtime alliance with Otsuka subsidiary Astex. — Amber Tong

  • Company: Evive Biotech and Acrotech Biopharma
  • Price: N/A
  • Peak sales estimate: N/A
  • Approval date: 11/16/2023

61. Ryzneuta

Active ingredient: efbemalenograstim alfa-vuxw

Indication: To treat neutropenia

Snapshot: Evive and Acrotech secured approval in November for Ryzneuta to decrease the incidence of febrile neutropenia in adults with non-myeloid malignancies who are receiving myelosuppressive cancer drugs. It’s the first innovative biologic independently developed by Evive Biotech, a subsidiary of China-based Yifan Pharmaceutical. — Zachary Brennan

  • Company: SpringWorks Therapeutics
  • Price: $29,000 per 30-day supply
  • Peak sales estimate: $1.66 billion – Bloomberg analyst consensus
  • Approval date: 11/27/2023

62. Ogsiveo

Active ingredient: nirogacestat

Indication: To treat adults with progressing desmoid tumors who require systemic treatment

Snapshot: Ogsiveo was approved in November following a three-month delay after regulators said they needed more time to review responses they requested from the company. The drug was licensed from Pfizer, which had tested it in a Phase I trial across solid tumor types. It was later discovered that desmoid patients “responded dramatically,” and patients advocated for enrollment in a Phase II study conducted by the National Cancer Institute, according to SpringWorks. — Nicole DeFeudis

  • Company: Novartis
  • Price: $550,000 per year
  • Peak sales estimate: More than $3B for all indications in late-stage development – Novartis
  • Approval date: 12/05/2023

63. Fabhalta

Active ingredient: iptacopan

Indication: To treat paroxysmal nocturnal hemoglobinuria

Snapshot: Fabhalta was approved to treat paroxysmal nocturnal hemoglobinuria, a rare disorder where red blood cells break up early. The twice-daily oral pill rivals AstraZeneca’s Soliris and Ultomiris infusions, which have been widely used for years and generated more than $5.7 billion in 2022 sales. However, Fabhalta carries a boxed warning for serious infections caused by encapsulated bacteria, and its use will be limited under a Risk Evaluation and Mitigation Strategy (REMS) program. — Nicole DeFeudis

  • Company: Vertex Pharmaceuticals and CRISPR Therapeutics
  • Price: $2.2 million
  • Peak sales estimate: $2.35 billion – Bloomberg analyst consensus
  • Approval date: 12/08/2023

64. Casgevy

Active ingredient: exagamglogene autotemcel

Indication: To treat sickle cell disease (SCD) in patients 12 years and older

Snapshot: Dec. 8 was an important day for sickle cell patients as the FDA approved two new gene therapies to treat the disease, including Casgevy, the first CRISPR treatment in the US. The decision capped decades of research and follows approvals in the UK in mid-November for sickle cell disease and transfusion-dependent beta thalassemia. Vertex will take the lead on manufacturing and commercialization and plans to open around 50 authorized treatment centers across the US, chief operating officer Stuart Arbuckle told Endpoints News. — Nicole DeFeudis

  • Company: bluebird bio
  • Price: $3.1 million
  • Peak sales estimate: $386 million – Bloomberg analyst consensus
  • Approval date: 12/08/2023

65. Lyfgenia

Active ingredient: lovotibeglogene autotemcel

Indication: To treat sickle cell disease (SCD) in patients 12 years and older

Snapshot: Lyfgenia was one of two gene therapies for sickle cell approved on Dec. 8, in addition to Vertex and CRISPR’s Casgevy. However, bluebird said it would price Lyfgenia nearly $1 million more than Casgevy, raising questions about access. The company also failed to secure an anticipated priority review voucher that it was hoping would bolster its cash reserves and instead turned to a combination of public and private financing. — Nicole DeFeudis

  • Company: Chiesi
  • Price: Chiesi spokesperson said the list price will be “competitively priced”
  • Peak sales estimate: N/A
  • Approval date: 12/18/2023

66. Filsuvez

Active ingredient: birch triterpenes

Indication: To treat wounds associated with dystrophic and junctional epidermolysis bullosa

Snapshot: Filsuvez is approved to treat wounds associated with junctional and dystrophic forms of epidermolysis bullosa, a rare skin disease characterized by chronic blistering and other wounds. It was rejected less than two years ago while in the hands of Amryt Pharma after regulators requested “additional confirmatory evidence of effectiveness,” according to the biotech. Chiesi bought the company for $1.25 billion last year. — Nicole DeFeudis

  • Company: Ionis/AstraZeneca
  • Price: “Consistent” with Onpattro and Amvuttra (around $450,000 per year)
  • Peak sales estimate: $1.57 billion – Bloomberg analyst consensus
  • Approval date: 12/21/2023

67. Wainua

Active ingredient: eplontersen

Indication: To treat hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN)

Snapshot: Wainua picked up approval in December as AstraZeneca and Ionis position it as an alternative to two of Alnylam’s drugs for hereditary transthyretin-mediated amyloid polyneuropathy, or ATTRv-PN. It will likely be compared most to Amvuttra, which, like Wainua, is an injectable drug. AstraZeneca and Ionis are also shooting for the larger ATTR-CM population, finishing enrollment in July. — Max Gelman

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Economic Trends, Risks and the Industrial Market

By a show of hands, I.CON West keynote speaker Christine Cooper, Ph.D., managing director and chief U.S. economist with CoStar Group, polled attendees…



By a show of hands, I.CON West keynote speaker Christine Cooper, Ph.D., managing director and chief U.S. economist with CoStar Group, polled attendees on their economic outlook – was it bright or bleak? The group responded largely positively, with most indicating they felt the economy was doing better than not.  

Four years ago, the World Health Organization declared COVID-19 a global pandemic, seemingly halting life as we knew it. And although those early days of the pandemic seem like a long time ago, we’re still in recovery from two of its major consequences: 1) the $4 trillion in economic stimulus that the U.S. government showered on consumers; and 2) the aggressive monetary policies that have created ripple effects on the industrial markets. 

Cooper began with an overview of the economic environment, which she called “the good news.” The nation’s GDP is strong, and the economy gained momentum in the second half of 2023 – we saw economic growth of 4.9% and 3.2% in Q3 and Q4 respectively — much higher than expected. “The reason is consumers,” Cooper said. “When things get tough, we go shopping. This generates sales and economic activity. But how long can it last?” 

Consumer sentiment continues to be healthy, and employment is good, although a shortage of workers could impact that moving forward. The U.S. added 275,000 jobs in January, far exceeding expectations. “The Fed raising interest rates hasn’t done what it normally does – slow job growth and the economy,” said Cooper. In addition, the $4 trillion given to keep households afloat during the pandemic has simply padded checking accounts, she said, as consumers couldn’t immediately spend the money because everyone was staying home, and the supply chain was clogged. The money was banked, and there’s still a lot of it to be spent. 

Cooper addressed economic risks and the weak points that industrial real estate professionals should be mindful of right now, including mortgage rates that remain at 20-year highs, stalling the housing market, particularly for new home buyers. Mid-pandemic years of 2020-2021 had strong home sales, driven by people moving out of the city or roommates dividing into two properties for more space and protection against the virus. Homeowners who refinanced in the early stages of the pandemic were fortunate and aren’t willing to list their houses for sale quite yet. 

“The housing market is a big driver of industrial demand – think furniture, appliances and all the durable goods that go into a home. This equates to warehouse space demand,” said Cooper. 

Interest rates on consumer credit are spiking and leading economic indexes are still signaling a recession ahead. Financial markets are indicating the same, with a current probability of 61.5% that we will be in a recession by 2025. However, Cooper said, while all signs point to a recession, economists everywhere say the same thing as the economy seemingly continues to surprise us: “This time is different.” 

Consumers are still holding the economy up with solid job and wage gains, yet higher borrowing costs are weighing on business activity and the housing market. Inflation has eased meaningfully but remains a bit too high for comfort. We’ve so far avoided the recession that everyone predicted, and the Federal Reserve appears ready to cut rates this year.  

For the industrial markets, the good news is that retailer corporate profits are beginning to bounce back after slowing in 2021 and 2022, with retail sales accelerating.  

A slowdown in industrial space absorption was reflected in all the key markets – Atlanta, Chicago, Columbus, Dallas-Fort Worth, Houston, the Inland Empire, Los Angeles, New Jersey and Phoenix – but was worst in the southern California markets, which have since been rebounding.  

“Supply responded to strong demand,” Cooper said. “In 2021, 307 million square feet were delivered, followed by 395 million in 2022. In 2023, we saw 534 million square feet delivered – that’s almost 33% higher than the year before.” 

The top 20 markets for 2023 deliveries measured by square feet are the expected hot spots: Dallas-Fort Worth (71 million square feet) leads the pack by almost double its follower of Chicago (37 million), then Houston (35 million), Phoenix (30 million) and Atlanta (29 million). Measured by share of inventory, emerging markets like Spartanburg, Pennsylvania, topped the list at 15 million square feet, followed by Austin (10 million), Phoenix and Dallas-Fort Worth (7 million), and Columbus (6 million). 

“Developers are more focused on big box distribution projects, and 90% of what’s being delivered is 100,000 square feet or more,” Cooper said. Around 400 million square feet of space currently under construction is unleased, in addition to the around 400,000 square feet that remained unleased in 2023. “Putting supply and demand together, industrial vacancy rate is rising and could peak at 6-7% in 2024,” she said. 

In conclusion, Cooper said that industrial real estate is rebalancing from its boom-and-bust years. Pandemic-related demands and accelerated e-commerce growth created a surge in 2021 and 2022, and the strong supply response that began in 2022 will continue to unfold through 2024. With rising interest rates putting a damper on demand in 2023, vacancies began to move higher and will continue to rise this year.  

“Consumers are spending and will continue to do so, and interest rates are likely to fall this year,” said Cooper. “We can hope for a recovery from the full effects of the pandemic in 2025.” 

This post is brought to you by JLL, the social media and conference blog sponsor of NAIOP’s I.CON West 2024. Learn more about JLL at or

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Pharma and biotech’s top R&D spenders in 2023: a $153B total with M&A as a focus

At a time when biotech is still counting its losses as a thaw gradually sets in after the long market winter, pharma has been on a tear. M&A took off…



At a time when biotech is still counting its losses as a thaw gradually sets in after the long market winter, pharma has been on a tear. M&A took off in Q4 as the industry’s biggest R&D spenders either rolled the dice on the back of their blockbuster bonanzas, were forced to address gaping holes in the pipeline in the face of looming patent expirations, or simply had no choice in the face of repeated setbacks.

Bioregnum Opinion Column by John Carroll

For some, it was all of the above.

As a result, Merck flipped into the lead position generally occupied by Roche with an M&A-inflated expense line for research. The companies joined a hunt for new drugs frequently focused on Phase III; premiums are in — heavy preclinical risks are out of favor. The majors followed some well-worn paths into immunology and oncology. And 2024 kicked off with a new round of buyouts and licensing deals.

The sudden end of Covid as a vaccine, drug and diagnostic market left the likes of Pfizer scrambling to convince investors that they had an exciting new plan. (It’s not working so far.) Eli Lilly has become one of the most valuable companies on the planet as obesity drugs go mainstream. Leaders like Takeda kept upping the ante on the R&D budget as the numbers frayed, with all but Pfizer and Bristol Myers Squibb — two of the most deeply off-balance biopharmas — spending more in 2023. Across the board, we saw $153 billion accounted for in R&D budget lines for last year — which would have registered as a record even without the sudden bolus of spending at Merck.

New, promising drugs at biotechs aren’t getting cheaper. And some of the blockbusters pharma has to cover as the patent cliff approaches will demand multiple replacement franchises.

The Big 15 have the money, desire and need to do much, much more in R&D. And all signs indicate that we’ll see more through 2024.

  • Merck
  • Roche
  • J&J
  • Novartis
  • AstraZeneca
  • Pfizer
  • Eli Lilly
  • Bristol Myers Squibb
  • GSK
  • AbbVie
  • Sanofi
  • Gilead
  • Takeda
  • Amgen
  • Novo Nordisk

1. Merck: The BD team is remaking the pipeline, and they are moving fast

  • R&D spending 2023: $30.5 billion
  • R&D spending 2022: $13.5 billion
  • Change: +125%
  • Revenue: $60.1 billion
  • R&D as a % of revenue: 51%
  • R&D chief: Dean Li
  • Ticker: $MRK — up 16% in the past year

The big picture: Merck moved up to the top of the list this year by bundling a mother lode of M&A and drug licensing deals into the R&D expense line. Otherwise, the top slot would have gone to Roche, the traditional top title holder in the R&D 15.

Merck has been parlaying its unchallenged position as number one in the PD-1 game with Keytruda — a drug that earned $25 billion last year but will face a loss of exclusivity as patents start to expire in 2028 — into a host of big deals in 2023. Keytruda, meanwhile, has cruised to 39 approvals, leaving Bristol Myers’ Opdivo in its wake.

Too much commercial success, though, doesn’t translate into unending praise. Analysts had been grumbling for some time that Merck wasn’t doing enough to diversify its pipeline bets. But that’s been changing.

Merck tallied $5.5 billion upfront for its Daiichi Sankyo deal — picking up rights to three ADCs in the move — along with the across-the-slate hikes in costs for clinical programs, bigger payrolls and benefits. There was another charge for the $11.4 billion that went to buying Prometheus and Imago. Prometheus accounted for $10.8 billion of that — one of the biggest deals that followed the $11.5 billion Acceleron buyout in 2021. With $690 million in cash for a group of partners that includes Moderna, Orna and Orion.

Merck kicked off the new year with a $680 million buyout of Harpoon Therapeutics, underscoring its enduring interest in the oncology market. And it’s leaving no popular stone unturned, capturing attention with its expressed interest in GLP-1 combos as the next generation of weight loss drugs takes shape.

Merck CEO Rob Davis also recently made it clear that the pharma giant can afford more $1 billion-to-$15 billion deals, making it a top candidate for more deals in 2024.

Merck’s firepower on the deals side, though, is needed after some deep wrinkles marred the pipeline plan, like the FDA’s back-to-back CRLs for chronic cough drug gefapixant. The data, however, never matched up to Merck’s rhetoric. Failures in Alzheimer’s and depression underscored Merck’s traditional ill fortunes in neuro.

Merck has a few years to plan for its next big thing. They show every sign of remaining focused on the big prize ahead.

2. Roche: 2023 was a tough year. Will 2024 be any better on the R&D side?

  • R&D spending 2023:  $16.1 billion/group — pharma and diagnostics (14.2 billion CHF)
  • R&D spending 2022: $16 billion/group (14.1 billion (CHF)
  • Change:
  • Revenue: $67 billion (58.7 billion CHF, -7% from 63.3 billion CHF in 2022)
  • R&D as a % of revenue: 24%
  • R&D chiefs: Hans Clevers (pRED), Aviv Regev (gRED), CMO Levi Garraway
  • Ticker: $RHHBY — down 4.8% in the past year

The big picture: It’s not easy being Roche. The behemoth has long had a near-omnivorous approach to R&D, buying up and down the pipeline at all stages with a big appetite for oncology ahead of neuro, ophthalmology and immunology. This year, it’s had to contend with the elimination of its Covid revenue, once a big player on the diagnostics side as testing soared during the pandemic. They’ve had to lower investors’ expectations of 2024 sales to an embarrassingly modest level and saw their stock price slide.

It’s surprising they have any growth, given the corresponding knockoff competition building for Lucentis and Esbriet, but you can’t play with market expectations. They’ll kill you every time you’re off.

Roche found some silver linings in the Vabysmo franchise and they’ve been a significant player on the M&A side, scoring the Carmot buyout for $3 billion after bagging Telavant for $7.1 billion back in October, paying a price for something Pfizer all but gave away to Roivant. James Sabry and the BD team, meanwhile, have kept up their globetrotting ways, uncorking a slate of deals for JP Morgan.

Sabry moved to global BD chief at Roche after winning his spurs at Genentech, and he’s been in the game for quite a long time. His résumé includes a stint as a biotech CEO. He’s the doyen of dealmakers and isn’t sitting on the sidelines. Hope grows eternal at Roche, and to keep it growing, Sabry has to stay busy.

“We have in total 12 NMEs that could potentially transition into a Phase III during this year,” CEO Thomas Schinecker told analysts hopefully during their Q4 call.

On this scale, Roche tends to do things on a wholesale basis. So when execs recently unveiled a pipeline review, they mapped 146 programs covering 82 new molecular entities. That can be hard to keep up with. If raw numbers like that were a good indicator of future success, though, Roche wouldn’t have these troubles.

It’s less difficult to follow the culls. That includes a slate of neurology drugs, with several axed from the oncology area. The write-offs include the longtime disappointment crenezumab, which had been partnered with AC Immune in Alzheimer’s. Roche recently handed back crenezumab as well as semorinemab after working with AC Immune for close to an R&D generation. Some analysts gave up long ago.

We’ve also been hearing complaints about a lack of upcoming pivotal clinical data to arouse enthusiasm. But Roche has two big R&D groups at work trying to counter those impressions, with gRED (Genentech) and pRED (the traditional Roche research group) at bat. They now have a straight-up GLP-1/GIP drug in the clinic for obesity, with oral therapies in the works alongside many others. It may be late to the obesity game with the Carmot buyout, but Roche still sees opportunities worth paying for.

Execs are promising to play a better R&D game, prioritizing their best assets and piling on resources. But Roche has always been willing to invest heavily in R&D. Now the company needs to see some clinical cards fall its way. This has not been a patient market.

3. J&J: Under new management, J&J doubles down on the innovative side of R&D. Can they still surprise us?

  • R&D spending 2023: $11.96 billion in meds
  • R&D spending 2022: $11.64 billion in meds
  • Change: Up 3%
  • Revenue: $54.7 billion (pharma side)
  • R&D as a % of spending: 21.8%
  • R&D chief: John Reed
  • Ticker: $JNJ —  up 5.3% in the past year

The big picture: J&J typically has weighed in heavy on R&D, particularly if you add its medtech work to the total. Even after splitting that out, though, it’s still in the top five, hoovering up large numbers of early-stage licensing deals while occasionally nabbing something major in the $1 billion-plus category.

Last year the pharma giant punted its consumer division, following the footsteps of many major industry outfits, and shut down its work in infectious diseases and vaccines. RSV, a highly competitive field now, went out the window with a host of smaller programs and alliances. Its major fields of interest zero in on oncology, immunology, cardio and retinal disorders. And they chipped in close to $2 billion to join the ADC hunt in January with its acquisition of Ambrx.

J&J earned a rep for out-of-the-box thinking in oncology under former oncology R&D chief Peter Lebowitz, striking a deal with China’s Legend that delivered an approved drug — Carvykti — and following up with a $245 million pact to gain worldwide rights to another CAR-T from CBMG, a low-profile Chinese biotech that erupted into mainstream view with its Big Pharma deal.

Now the big questions about J&J focus on its new leadership after Joaquin Duato moved into the CEO’s role in 2022 and John Reed — leaping into his third Big Pharma R&D posting in 10 years, following Roche and Sanofi — takes command of the global R&D side of the company.

They have plenty of motivation to hustle up major new approvals. Stelara — raking in more than $10 billion a year — will see its patent protection erode in the US in 2025, with Europe moving first this year. That will take a few big wins to cover.

But J&J has been making big promises for years. Just a few months ago, it touted 20 drugs in the pipeline that could fuel 5% to 7% growth through 2030. One of the prime candidates is a drug they picked up from Protagonist: JNJ-2113, an IL-23 they believe can bring in blockbuster revenue in immunology. J&J, though, is likely far from done when it comes to new deals. Oncology R&D has been changing rapidly in the wake of the Inflation Reduction Act, with researchers moving up OS as a primary initial focus in Phase III. And it’s going to take a behemoth effort to deliver on these numbers, with likely failures and shortfalls along the way.

Don’t look for J&J to cut R&D anytime soon. They have a big agenda.

4. Novartis: Another streamlining move is wrapping up as Novartis vows to get back to basics in R&D — again

  • R&D spending 2023: $11.37 billion
  • R&D spending 2022: $9.17 billion
  • Change: Up 24%
  • Revenue: $45.44 billion
  • R&D as a % of revenue:  27%
  • Development chief: Shreeram Aradhye, NIBR chief: Fiona Marshall
  • Ticker: $NVS — up 31% in the past year

The big picture: Novartis CEO Vas Narasimhan has been crystal clear about the Big Pharma’s M&A strategy. He’s sticking with the industry sweet spot now in favor: picking up late-stage assets below the $5 billion range. A few weeks ago, that led Novartis to MorphoSys, where they have been partnered for years while distancing themselves from rumors of a pricey Cytokinetics play.

And it springs right off another $3 billion acquisition — for Chinook — that went straight to positive Phase III data for the kidney drug atrasentan, which likely wasn’t much of a surprise inside Novartis.

These days, Narasimhan and Novartis are all about focus. They want to make a deeper impact where they emphasize their priorities — cardio, immunology, neuroscience and oncology. And they also want to be leaders where they are centered, slashing oncology while emphasizing at every opportunity that they jumped out front in radioligands, now a hot commodity in R&D.

Lest anyone forget, Novartis was a pioneer in autologous CAR-T and has held on as it slowly works through all the challenges a cutting-edge technology can inspire.

Narasimhan had been five years before the mast as CEO, after being promoted from development chief, and he’s revising a pipeline strategy away from something he describes now as akin to everything everywhere all at once. Downsizing in 2023 was the big focus, dropping programs, reassigning scientists and promising a swifter pace — a never-ending problem in Big Pharma land. Narasimhan has also been pushing “seamlessness,” projecting a new era of cooperation among scientists and sales.

There’s nothing new about streamlining at Novartis, though. Narasimhan had a billion dollars of cuts in mind back in the spring of 2022. And periodically, the company has been well-known for going in and ironing out budgets. Changes have included an exit for development chief John Tsai, now a biotech CEO, who was replaced by Shreeram Aradhye. Fiona Marshall took the helm at NIBR in the fall of 2022, taking the place of Jay Bradner, who left and later wound up running R&D at Amgen.

The recent cleanup at Novartis included the end of the deal for BeiGene’s PD-1, an area that proved enormously frustrating to Novartis. Their TIGIT pact ended last summer. Phase II for GT005, a gene therapy it picked up in the $800 million Gyroscope buyout, didn’t end well. That program got the axe. And their anti-TGFß antibody, picked up in a small deal with Xoma nine years ago, failed after execs once billed it as a high-risk, high-reward play. Other setbacks include Adakveo, which faced global regulatory challenges following the failure of the Phase III confirmatory study. At the beginning of this year, there was a snafu in Phase III for ligelizumab, once billed as a top asset for peanut allergies.

Warning clouds have also formed around their top-selling drug Entresto, as Novartis fights a battle against the IRA and price negotiations.

The CEO, though, has been able to transition while the stock price was headed up, with a few big drugs driving revenue growth as a struggling Sandoz finally got the heave-ho in a spinout. Their franchise drug Kisqali, for example, is now billed as a $4 billion earner at the peak. As a result, their story has played well on Wall Street. Investors want to see the money and the trajectory. R&D follows sales in priority when it comes to the majors.

5. AstraZeneca: Pascal Soriot never takes defeat lying down. And that stubborn attitude has delivered big dividends as another big R&D test takes shape

  • R&D spending 2023: $10.93 billion
  • R&D spending 2022: $9.76 billion
  • Change: Up 12%
  • Revenue: $45.8 billion
  • R&D as a % of revenue: 24%
  • R&D chiefs: Sharon Barr (biopharmaceuticals); Susan Galbraith (oncology)
  • Ticker: $AZN — up 1.8% in the past year

The big picture: Back in 2018, AstraZeneca reported R&D expenses just under $6 billion. In the past five years, that big line item has grown 85%, and investors have seen the stock price grow 56%.

The R&D leaders at AstraZeneca have changed, but CEO Pascal Soriot has become a longtime fixture at the company. During his stint he took the weakest pipeline in biopharma and turned it into one of the strongest, building a slate of blockbuster oncology franchises while building a research machine based in Cambridge, UK, that consumes about $1 out of every $4 in revenue. He bet the ranch on Enhertu and won, with some analysts bullishly projecting peak sales that will break $10 billion. And he’s kept many of the promises he had to fire out to investors to keep an unwanted Pfizer takeover at bay in the way back when.

So what’s next?

That’s a question that’s vexing quite a few analysts. AstraZeneca is a restless player and the company takes a lot of chances — which means it racks up a lot of setbacks.

A major initiative aimed at protecting its revenue involves its legal fight against the IRA, which AstraZeneca has so far lost. Its next big ADC with Daiichi Sankyo, Dato-DXd, has sparked a running debate on its potential approval and some analysts have doubted if it can live up to the hype following weak PFS results for the TROP2 ADC. Last summer an early-stage GLP-1 went down in flames, unable to take the heat in a kitchen currently controlled by the commercial chefs at Novo Nordisk and Eli Lilly. Lokelma, picked up in a 2015 buyout, got hit when R&D decided to quash two Phase III studies, denting once-big hopes for blockbuster status. And Soriot has recently been forced to finally give up on one old failure when he finally punted roxadustat’s US rights.

Soriot, though, is a weathered player when it comes to setbacks. Every loss is an opportunity to do better the next time, and no one can be more stubborn. You could see that play out over Covid when its vaccine came in for some undue criticism that blighted its impact in the face of the mRNA stars. That spurred some angry responses as execs dug in. But there was an unexpected upside. The giant didn’t have to readjust as the Covid market went pfffffft.

Their next step: A couple of months ago AstraZeneca touted its new vaccine platform, buying Icosavax for $838 million in cash while contributing an RSV vaccine to the pipeline — a field where GSK has made major headway — and a virus-like particle platform that the company intends to build on.

Volrustomig, a PD-1/CTLA-4 bispecific antibody, has been accelerated into Phase III, with Soriot claiming a leadership spot in bispecifics: “Our portfolio of bispecifics has the potential to replace the first-generation checkpoint inhibitors across a range of cancers.”

And that GLP-1 fail? Last November AstraZeneca paid $185 million to gain a Phase I GLP-1 drug out of China’s Eccogene. And now they’re mapping combo studies with some of their other drugs in a play at creating the next wave of obesity therapies with an edge.

Word in biopharma is that Soriot has been devoting a considerable amount of face time to China, where he committed the company years ago. That’s another one of those market promises that has seen plenty of ups and downs. But Soriot tends to win the big gambles more than he loses, and in this industry, seeing it through can be a major long-term advantage.

6. Pfizer: What the hell happened to the Covid king?

  • R&D spending 2023: $10.57 billion
  • R&D spending 2022: $11.4 billion
  • Change: -7.3%
  • Revenue: $58.5 billion (down 42% from $100.3 billion)
  • R&D as a % of revenue: 18%
  • R&D chief: Mikael Dolsten
  • Ticker: $PFE — down 29% in the past year

The big picture: There was one brief, shining moment — or two — when Pfizer could seemingly do no wrong. It had taken a leading role in breaking through scientific barriers to create a new Covid vaccine in record time, harvested a bumper crop of cash and CEO Albert Bourla was the darling of the world’s favored pharma industry.

That was then.

Now, Bourla and his team are having a tough time convincing Wall Street that the company can do even simple things right. They paid $43 billion to bag Seagen and mount a major new campaign on the cancer front, but its stock has been blighted and the focus turned to cost-cutting as revenue plunged. There was fresh humiliation when Roivant flipped a drug it had grabbed from Pfizer for lunch money and sold it to Roche for $7.1 billion a year later. And Pfizer has lost the narrative in convincing investors it can get back to growth.

That somewhat hapless rep was burnished considerably when Pfizer reported that its first try at an oral GLP-1 obesity drug had flopped. It’s still working to move the dial in the hottest new field in pharma, but so is a long list of rivals. Instead of spurring renewed faith in Pfizer, the obesity play turned into another example of getting it wrong, and the focus at Pfizer shifted squarely to downsizing and cost-cutting in acknowledgment of the new reality that set in.

Bourla, though, is committed to pushing the story that a new period of growth lies ahead. And it’s not proving easy.

At the end of February, Pfizer made its best pitch for oncology, underscoring plans to seize the leadership role in genitourinary and breast cancer while making promises for eight-plus possible blockbusters in the next six years. R&D promises, though, are easy to make and hard to keep. Right now, the clarion call in pharma is “show me the money.”

With Covid and the mRNA revolution forgotten like last season’s hit show, there’s an enormous gap now that will be devilishly hard to bridge. But don’t expect anyone at Pfizer to stop trying anytime soon.

7. Eli Lilly: Built for the long term, Lilly’s day has arrived — and they don’t want to let go

  • R&D spending 2023: $9.31 billion
  • R&D spending 2022: $7.2 billion
  • Change: +30%
  • Revenue: $34.1 billion
  • R&D as a % of revenue: 27%
  • R&D chief: Dan Skovronsky
  • Ticker: $LLY — up 126% in the past year

The big picture: Historically, Eli Lilly has been known as a ponderously slow pharma outfit that often slowly cruised its way into Phase III squalls. But that view is so 2017. In 2024, Lilly has rebranded itself as the Big Pharma engine that could, and did, blow out expectations. And if it’s still not quite as nimble as some analysts might like, its ability to deliver in massively expensive late-stage studies for drugs aimed at big populations has made it a darling of quite the investor crowd.

Lilly, for example, was thwarted at getting an accelerated approval for its Alzheimer’s med, but that didn’t really cut expectations, with blockbuster peak sales projections — even as Biogen/Eisai’s Leqembi suffers from dimming prospects as their high hopes are lowered by the reality of limited sales in the face of limited efficacy.

That pales, though, in comparison to the bright rainbow that’s emerged in obesity. Lilly continues to work up manufacturing capacity to meet demand for its new obesity version of tirzepatide, the GLP-1/GIP drug building up the diabetes franchise, where neither of the two leaders has been able to meet a seemingly limitless demand.

Lilly attracted considerable attention for its vow to build out manufacturing capacity ahead of Phase III data for its next-gen oral version, orforglipron, while clearly so unhappy about Novo’s decision to muscle in and snap up Catalent that CEO Dave Ricks is grousing about the antitrust implications of their rival’s move. Lilly, though, has bragging rights to solid pivotal data in a market that is nowhere close to saturation point.

Like a lot of the big spenders on the list, Eli Lilly has been hunting new immunology drugs and plunked down $2.4 billion for Dice last summer. That was part of a full slate of acquisitions, including a pair of small ADC companies. Following yet another hot trend, there was a $1.4 billion deal for Point, which put them into radiopharmaceuticals.

Lilly nabbed two new drug approvals last year as it waited on the 2 big franchises in obesity and Alzheimer’s. That’s a testament to the progress that Dan Skovronsky spurred after the global player made him R&D chief 6 years ago. Eli Lilly execs still may not always be first, in an industry where first can be tremendously important to commercialization. But they’ve been right where it counts big in drug development, and it will take a therapeutic earthquake to alter that perception anytime in the near term.

8. Bristol Myers Squibb: A rough year spurs a cut in R&D spending and some major late-stage R&D deals

  • R&D spending 2023:  $9.299 billion
  • R&D spending 2022: $9.5 billion
  • Change: -2%
  • Revenue: $45 billion
  • R&D as a % of revenue: 20.6%
  • Development chief: Samit Hirawat; Research chief: Robert Plenge
  • Ticker: $BMY — down 18% in the past year

The big picture:  This is a terrible time to try and explain why your Big Pharma company has structural issues that flattened or eroded sales revenue. Pfizer understands that and Bristol Myers got a bad taste of it as its shares slid 18% in the last year.

In both cases, the CEOs stepped up with a transition plan. The companies did some deals, but the late-stage stuff wasn’t cheap. And in Bristol Myers’ case, a new CEO was able to draw a line between its aging franchises and the new arrivals on the market, which saw some growth. The company line now: Just wait for the big pipeline hits to come and give us some time to weather the decline of these legacy drugs and you’ll love what you see.

Investors may not be cheering, but Bristol Myers’ stock did get some traction out of it in the last few weeks.

It was clear well before 2023 arrived that Bristol Myers understood it was facing some of those dreaded headwinds. That 2% drop in R&D spending highlighted the tight rein on spending for what remains a top 10 player in the pharma R&D world. Major figures in R&D, headed by Rupert Vessey, exited the company — in Vessey’s case, later making the flip to biotech at Flagship. And there was an unusual spat with Dragonfly after the pharma giant walked away from its $650 million investment.

New CEO Chris Boerner spotlighted the immediate strategy at hand: M&A. Mirati and KRAS came their way for $5.8 billion. RayzeBio happily landed a premium on top of the premium they had just scored in an IPO, as Bristol Myers followed rivals into radiopharmaceuticals. The $14 billion Karuna buyout put them into a late-stage race on Alzheimer’s, another R&D category that’s been enjoying a renaissance some years after pharma fled the scene.

Boerner’s bottom line in the Q4 review is that the company will steer more into bolt-on plays — rather than big buyouts — and licensing deals like the SystImmune alliance. That sets the stage for a “transition” period that will last until 2028, four long years ahead, when it’s promising “top-tier” results. It will also be looking at lower-priced competition for Opdivo.

Even before 2028, though, BMS will start losing patent protection on Eliquis. They’ve already begun price negotiations with Medicare. And Eliquis earned $12.2 billion in 2022, making it their number-one franchise. That’s left Bristol Myers and Pfizer, both under huge pressure to perform and do more late-stage deals, backing a full-court press in the courts to keep generics at bay.

Bristol Myers has had an active dealmaking arm for years, including in the wake of its big $74 billion buyout of Celgene, which also delivered Vessey to the pharma giant. That was just five years ago after Celgene had fallen on some troubled times. Celgene had been a standout in the licensing field, known for sampling a wide variety of drug plays in the industry pipeline. One of Bristol’s big failures, though, was ceding the high ground in PD-1 to Merck’s Keytruda, which has been buoying its rival for years. Bristol needs major drug franchises to make a difference in this world, and any future setbacks on the leading drugs it’s been buying now will not be welcome by investors.

There is a path forward for Bristol, of course, even as it vows to pay down debt. But it’s fairly narrow, and this field is known for some treacherous results.

9. GSK: After picking up some badly-needed revenue steam, what’s next for R&D?

  • R&D spending 2023: $7.9 billion (£6.22 billion)
  • R&D spending 2022: $7 billion (£5.5 billion)
  • Change: +13%
  • Revenue: $39 billion (£30.3 billion)
  • R&D as a % of revenue: 20.5%
  • R&D chief: Tony Wood
  • Ticker: $GSK — up 28% in the past year

The big picture: Tony Wood is still shy of his second anniversary as the CSO at GSK, but with an RSV vaccine riding high as a new blockbuster franchise and Shingrix looking every bit the long-distance franchise player GSK needs, he has a reassuring revenue foundation to work with. ViiV’s steady work in HIV — where GSK is a majority owner — also offers a confidence-building revenue stream. And the departure of the consumer unit is well into the rearview mirror now.

Its stock has done well, too, up 28% in the past year.

That’s quite a changed picture from the early days of his predecessor, Hal Barron, who came in with deep oncology experience and a big need to demonstrate a broad-based pipeline reorganization to overcome a well-earned rep for underperformance. Wood’s first moves in R&D were largely defensive, giving up some major alliances — such as a partnership with Adaptimmune — that looked shaky.

GSK has made a lot of early bets, and the risks involved naturally portend that many of its deals won’t survive. You can see that in play right through its recent decision to dump a pair of Vir partnerships in infectious diseases.

In their place, GSK has been inking major new development deals with the likes of China’s Hansoh, for ADCs. Oncology, though, is still only a small performer overall. And it’s been a focus for a while.

GSK spent a billion dollars upfront to bag a mid-stage asthma drug at Aiolos in a rare M&A deal. There was also the $2 billion Bellus buyout last fall, with an eye to creating a new franchise for chronic cough. But there’s been a notable absence of any splashy deals at GSK, with a reorg in research that offers GSK’s latest take on improving efficiency.

We’ll see how that goes.

In the meantime, GSK is doing what it can to stir up some excitement for late-stage drugs like depemokimab (again in asthma), camlipixant (from Bellus) as well as the antibiotic gepotidacin for UTIs/gonorrhea. It’s an uphill fight, though, without much megablockbuster razzmatazz built in. But GSK is a careful player.

After getting stuck with the rep for having one of the worst pipelines in pharma, though, reliable and steady progress with a high-profile launch in RSV will suit just fine. At least for now. It’s likely that investors will keep pressing for something big in Phase III, and that could cost CEO Emma Walmsley a considerable amount of BD money.

10. AbbVie: The slow-motion collapse of Humira keeps them focused on the bottom line while growing R&D spending

  • R&D spending 2023: $7.67 billion
  • R&D spending 2022: $6.51 billion
  • Change: Up 18%
  • Revenue: $54.3 billion
  • R&D as a % of revenue: 14%
  • CMO: Roopal Thakkar
  • Ticker: $ABBV — up 18% in the past year

The big picture: As Rick Gonzalez finishes his final run as CEO, he’s able to look back on a year that saw AbbVie complete its revamp period as the long-awaited — long, long-awaited — arrival of generic Humira bites into its old cash cow.

The great split at Abbott that created AbbVie set up a scenario where the company would pull out every stop to milk Humira for every conceivable dollar possible, delivering mega-returns while Gonzalez became the poster child of patent reform. The bottom line for AbbVie’s team: What’s repeated waves of congressional criticism with the stock price on the line?

Now AbbVie is able to boost expected revenue on the two big drugs developed on Gonzalez’s watch — Skyrizi and Rinvoq — with two new acquisitions to feed future sales projections. The buyout of Botox created a new, highly reliable franchise for AbbVie’s commercial team to lean on.

AbbVie is skilled at acquiring and building revenue. It had its eyes set on the ADC drug Elahere when it acquired ImmunoGen for $10 billion. Initially approved in 2022 for ovarian cancer, the drug is now being positioned for earlier lines of therapy.

Less than a week after the ImmunoGen deal was announced, AbbVie was back for a late-stage acquisition with the $8.7 billion for Cerevel’s neuro play. The deal will bring in clinical-stage assets for schizophrenia, Parkinson’s and dementia, as CNS moves back into a warmer phase in Wall Street circles. Both buyouts underscore Big Pharma’s considerable appetite for new products, with premiums in play for de-risked drug programs.

Gonzalez’s departure barely caused a murmur on the markets, which is a testament to his success in delivering for shareholders a secure, long-term rebuild. His legacy is a company with a ruthless rep for shepherding drug revenue while building a big interest in curtailing patents for pharma. But looking only at the numbers, he proved the winner at the company as the game was played during his tenure.

11. Sanofi: Paul Hudson is still out to make a great first impression in R&D

  • R&D spending 2023: $7.09 billion (6.509 billion euros)
  • R&D spending 2022: $7.08 billion (6.503 billion euros)
  • Change:  flat
  • Revenue: $41.3 billion (37.9 billion euros)
  • R&D as a % of revenue: 17.1%
  • R&D chief: Houman Ashrafian
  • Ticker: $SNY — up 2.8% in the past year

The big picture: When Paul Hudson showed up in San Francisco for JP Morgan in January, ready to talk up plans for the road ahead, he noted: “It feels like a lot longer than four years that we’ve been on this journey.”

But Hudson has always been more comfortable sounding like a newly-coined CEO, plotting a turnaround. And in the last few months, he’s played every card in that deck. The announcement late last year that Sanofi is bumping its R&D budget is central to that theme, though the news of its impact on profitability led to a rout of the stock price. And he delights in spotlighting late-stage assets, even though a slate of his early bets failed or have yet to prove themselves.

In what is now standard in pharma, Hudson made what he could out of the news he was spinning out the consumer division. Again, though, investors were less than thrilled by the gambit.

This time around the PR track, Hudson has boasting rights to the recently approved RSV drug Beyfortus, which comes with some big peak sales projections from Jefferies and much, much less from others. We’ll know soon enough if this is a winner or the latest disappointment at Sanofi. And, as always, there’s the Sanofi touchstone: Its megablockbuster Dupixent, which the pharma giant was able to partner on with Regeneron years ago — keeping the franchise fresh and expanding. Dupixent is the cash cow that gives Sanofi the financial strength needed to move ahead.

And that means there’s capacity for more dealmaking.

Not long after the San Francisco appearance, Hudson followed up on his M&A assurances with a $1.7 billion drug buyout, carving out a Phase II drug for a rare disease called alpha-1 antitrypsin deficiency, or AATD. It fits right into the zone for 2024, where pharma can only get positive attention for something within sight of an approval.

Like others on this list, Sanofi’s R&D rep will ultimately rest on its ability to deliver on the 12 would-be blockbusters the company is betting on. That includes three “products in a pipeline“: amlitelimab, frexalimab and SAR441566 (oral TNFR1si). They’re followed by tolebrutinib, lunsekimig, rilzabrutinib, an anti-TL1A in IBD, an IRAK4 degrader and itepekimab for COPD.

Behind it all, Hudson has also been promising to make Sanofi a leader in AI-assisted pharma operations. Sanofi, though, has been promising a makeover in innovation for well over a decade and has done nothing to prove it’s worked beyond staying on track with the megablockbuster it got from Regeneron. One breakout franchise delivered on Hudson’s watch would change that in a heartbeat.

We’re waiting.

12. Gilead: The CEO gambled on big innovation — and often lost. But the wagers keep coming

  • R&D spending 2023: $5.72 billion
  • R&D spending 2022: $4.98 billion
  • Change: +14.6%
  • Revenue: $27.1 billion
  • R&D as a % of revenue: 21%
  • CMO: Merdad Parsey
  • Ticker: $GILD — down 5.3% over the past year

The big picture: Daniel O’Day jumped into the CEO job at Gilead five years ago and hit the ground running. He hasn’t stopped, even though some of his biggest bets have run into brick walls.

That was apparent weeks ago with the news that Gilead would ice its work on blood cancer involving magrolimab, the CD47 drug picked up in a $5 billion buyout back in 2020. Their mid-stage work on solid tumors ground to a halt shortly after.

Rehashing and refocusing their deal with Arcus, putting in significantly more money while axing one of the Phase IIIs, didn’t help.

Gilead’s rep was built around HIV, where it has remained dominant, though more than a bit taken for granted. The old regime’s follow-up — after a cloudburst of cash for curing hep C that quickly dried up — was to buy out Kite and take a pioneering position in CAR-T, which hasn’t lived up to the financial hype that attended its arrival, despite the clear scientific innovation it brought to the field.

The stock was hammered hard in January after Trodelvy — acquired in the 2020 Immunomedics buyout, which achieved blockbuster status last year — failed a Phase III in second-line lung cancer.

But when you raise doubts and see your stock sinking, counter with a late-stage buyout. That’s clearly what O’Day had in mind when he plunked down more than $4 billion to buy CymaBay after the biotech unveiled late-stage data on seladelpar. Gilead bought a would-be blockbuster with a PDUFA date. And that’s a sign of some desperation at a company that badly needs a breakout.

13. Takeda: Moving up another notch on the top 15, as profitability wobbles, Takeda execs are still reaching for the golden ring in R&D

  • R&D spending 2023: $4.93 billion
  • R&D spending 2022: $4.49 billion
  • Change: +10%
  • Revenue: $29.54 billion
  • R&D as a % of revenue: 17%
  • R&D chief: Andy Plump
  • Ticker: $TAK — down 8.4% in the past year

The big picture: Takeda has been aggressively taking chances in R&D right from the time CEO Christophe Weber and R&D chief Andy Plump teamed up to remake the aging Japanese pharma company into a global drug player back in 2015. That meant steadily upping the ante in R&D — now up another slot in this year’s rankings — and investing in deals like the Shire buyout, which gave Plump his base in the Cambridge/Boston hub, along with a big stake in rare diseases.

For Takeda, that mission meant a broad effort to develop a major pipeline, from collaborations through Phase III. More recently, it’s been about concentrating their new work around a pair of key deals, particularly the $4 billion acquisition of Nimbus’ TYK2. It likely wasn’t much of a surprise, but their drug — which also has a $2 billion rider for milestones — cleared a Phase IIb hurdle in psoriatic arthritis.

For Takeda, it’s a clear indication of just how popular it is these days for pharma players to zero in on late-stage therapies in search of relatively near-term approvals.

Want more evidence of that?

Takeda bet $400 million in cash and more than a billion dollars in milestones to gain rights to Hutchmed’s fruquintinib and then was rewarded with an approval for treatment-naive cases of colorectal cancer in the fall. And they demonstrated its continued appetite in the rare disease space with the recent $300 million deal for Protagonist’s late-stage drug rusfertide, designed to treat a rare blood disease called polycythemia vera (PV).

The risks it’s taken on have been readily apparent to Takeda’s leaders, with its decision to drop Exkivity after flunking the Phase III NSCLC confirmatory trial, a Phase II fail for its key metachromatic leukodystrophy program, as well as a decision to drop Theravance as a partner after a seven-year alliance. The late-stage setbacks cost Takeda a $770 million write-down. Add in a loss of exclusivity for Vyvanse in 2023 — a $3 billion blockbuster in fiscal 2022 — and you have the outlines of unsteady performance for the pharma player, with Weber promising to do better in the near term.

Takeda is unusual in the Big Pharma world for winding up its fiscal year at the end of March. In order to do an apples-to-apples comparison, they prepared a summary of their R&D expenses and revenue for all of 2023 for Endpoints News.

14. Amgen: Capitalizing on a history of striking high-profile deals, Amgen stays in the spotlight

  • R&D spending 2023: $4.8 billion
  • R&D spending 2022: $4.4 billion
  • Change: Up 9%
  • Revenue: $28.2 billion
  • R&D as a % of revenue: 17%
  • R&D chief: Jay Bradner
  • Ticker: $AMGN — up 18% over the last year

The big picture: Amgen is a considerable distance from spending on research like the top 10 players in our R&D 15, but it frequently finds ways to box competitively in the biggest heavyweight category. It had done that with KRAS, taking a legit scientific advance that couldn’t quickly move the dial in a major way on the commercial side. That happens a lot in oncology. And now it’s in the spotlight with an obesity drug — branded as MariTide now — with hopes to take on the likes of Eli Lilly and Novo Nordisk.

The chutzpah originates with longtime CEO Bob Bradway, who has parlayed his Wall Street cred as a former banker at Morgan Stanley into major league status with a savvy understanding of the numbers and investors. He skillfully navigated the $28 billion Horizon buyout last year, bagging a lineup of commercial therapies as the company looks for the approaching patent cliff on Enbrel, a reliable blockbuster that has kept the revenue flowing in.

Amgen may not do a lot in M&A or Phase III, but what it does do, it does with style.

To complete the Horizon deal, Bradway had to orchestrate a deal with the FTC to skirt its objections to price bundling, which essentially leaves the pharma company on commercial probation with regular reporting to the federal agency. That took skill and boldness while maintaining the CEO’s rep for delivering on the bottom line. Its stock is up 18% over the past year.

Analysts will be watching carefully to see how Jay Bradner does in the top R&D post after the Harvard prof-and-former-NIBR chief assumes the seat of David Reese, now chief technology officer. Reese seems truly energized in his new role heading up tech, and Bradner is a die-hard research enthusiast who loves nothing better than jumping into conversations about the details of target degeneration.

Amgen is all about message.

15. Novo Nordisk: The longtime diabetes franchise player has a breakout run going in obesity — with vows to stay in front

  • R&D spending 2023: $4.7 billion (32.4 billion Danish Krone)
  • R&D spending 2022: $3.5 billion (24 billion Danish Krone)
  • Change: 34%
  • Revenue: $22 billion (232.2 billion Danish Krone)
  • R&D as a % of revenue: 14%
  • R&D chief: Marcus Schindler
  • Ticker: $NOVO — up 87% in the past year

The big picture: R&D spending as a percentage of sales has edged up a bit in the last few years, but the key driver here is GLP-1, where Novo has capitalized on its first-in-class leadership position in obesity. After decades spent in the shadow of chronic R&D failure, safety issues and a recent swarm of largely ineffective drugs, the obesity field is crushing it. That has swelled sales revenue as semaglutide glowed, so Novo’s research spending has boomed at a fast pace.

Now that the good times are rolling, and Novo already has a well-earned rep as a realistic and committed player in diabetes, which didn’t come cheap or easy, the new player on the R&D 15 is promising to stay out front — no easy task with Eli Lilly gunning for it. Novo has been snapping up new obesity tech at a furious pace, determined to stay out front.

Its one limiting factor here has been manufacturing capacity. Novo can’t satisfy the demand for a drug that is now a staple of public conversation, as the field gets a boost from a wide range of celebrities, including Oprah Winfrey. That’s marketing you could buy, but don’t have to. It’s coming for free.

With uncharacteristic bravado, Novo doubled down by striking a deal to acquire the global CDMO giant Catalent for $16.5 billion, and Lilly has been fuming about the antitrust aspects as CEO Dave Ricks complains that worldwide manufacturing capacity has either been maxed out or is not easily converted from its existing uses.

Novo’s commitment to growing R&D has international implications that far exceed the limits of its home country of Denmark, extending to hubs in Oxford, Seattle and Beijing. Most recently, Novo has committed to boosting its Boston-area research hub. And it’s likely to remain a key player in its dominant fields — unless some other tech can topple the megablockbuster that is remaking this company.

Novo may be at the end of this list in terms of R&D spending, but it has overachieved with its success for semaglutide. It has the capacity to do more and should continue to climb for several years to come as it makes a case for continued growth.

Postscript: Regeneron, with $4.44 billion in research spending — up 23% over $3.6 billion in 2022 — deserves an honorable mention in the competitive 16th spot. This year, Regeneron expects R&D spending to top up at or close to $5 billion. The company’s value has swollen on the success of its high-profile founders, Len Schleifer and George Yancopoulos, who continue to build the company — hitting a market cap in excess of $100 billion with the stock up 29% over the past year. Regeneron will likely find its way into the top 15 at some point, and we’ll be watching for it.

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Chronic stress and inflammation linked to societal and environmental impacts in new study

From anxiety about the state of the world to ongoing waves of Covid-19, the stresses we face can seem relentless and even overwhelming. Worse, these stressors…



From anxiety about the state of the world to ongoing waves of Covid-19, the stresses we face can seem relentless and even overwhelming. Worse, these stressors can cause chronic inflammation in our bodies. Chronic inflammation is linked to serious conditions such as cardiovascular disease and cancer – and may also affect our thinking and behavior.   

Credit: Image: Vodovotz et al/Frontiers

From anxiety about the state of the world to ongoing waves of Covid-19, the stresses we face can seem relentless and even overwhelming. Worse, these stressors can cause chronic inflammation in our bodies. Chronic inflammation is linked to serious conditions such as cardiovascular disease and cancer – and may also affect our thinking and behavior.   

A new hypothesis published in Frontiers in Science suggests the negative impacts may extend far further.   

“We propose that stress, inflammation, and consequently impaired cognition in individuals can scale up to communities and populations,” explained lead author Prof Yoram Vodovotz of the University of Pittsburgh, USA.

“This could affect the decision-making and behavior of entire societies, impair our cognitive ability to address complex issues like climate change, social unrest, and infectious disease – and ultimately lead to a self-sustaining cycle of societal dysfunction and environmental degradation,” he added.

Bodily inflammation ‘mapped’ in the brain  

One central premise to the hypothesis is an association between chronic inflammation and cognitive dysfunction.  

“The cause of this well-known phenomenon is not currently known,” said Vodovotz. “We propose a mechanism, which we call the ‘central inflammation map’.”    

The authors’ novel idea is that the brain creates its own copy of bodily inflammation. Normally, this inflammation map allows the brain to manage the inflammatory response and promote healing.   

When inflammation is high or chronic, however, the response goes awry and can damage healthy tissues and organs. The authors suggest the inflammation map could similarly harm the brain and impair cognition, emotion, and behavior.   

Accelerated spread of stress and inflammation online   

A second premise is the spread of chronic inflammation from individuals to populations.  

“While inflammation is not contagious per se, it could still spread via the transmission of stress among people,” explained Vodovotz.   

The authors further suggest that stress is being transmitted faster than ever before, through social media and other digital communications.  

“People are constantly bombarded with high levels of distressing information, be it the news, negative online comments, or a feeling of inadequacy when viewing social media feeds,” said Vodovotz. “We hypothesize that this new dimension of human experience, from which it is difficult to escape, is driving stress, chronic inflammation, and cognitive impairment across global societies.”   

Inflammation as a driver of social and planetary disruption  

These ideas shift our view of inflammation as a biological process restricted to an individual. Instead, the authors see it as a multiscale process linking molecular, cellular, and physiological interactions in each of us to altered decision-making and behavior in populations – and ultimately to large-scale societal and environmental impacts.  

“Stress-impaired judgment could explain the chaotic and counter-intuitive responses of large parts of the global population to stressful events such as climate change and the Covid-19 pandemic,” explained Vodovotz.  

“An inability to address these and other stressors may propagate a self-fulfilling sense of pervasive danger, causing further stress, inflammation, and impaired cognition in a runaway, positive feedback loop,” he added.  

The fact that current levels of global stress have not led to widespread societal disorder could indicate an equally strong stabilizing effect from “controllers” such as trust in laws, science, and multinational organizations like the United Nations.   

“However, societal norms and institutions are increasingly being questioned, at times rightly so as relics of a foregone era,” said Prof Paul Verschure of Radboud University, the Netherlands, and a co-author of the article. “The challenge today is how we can ward off a new adversarial era of instability due to global stress caused by a multi-scale combination of geopolitical fragmentation, conflicts, and ecological collapse amplified by existential angst, cognitive overload, and runaway disinformation.”    

Reducing social media exposure as part of the solution  

The authors developed a mathematical model to test their ideas and explore ways to reduce stress and build resilience.  

“Preliminary results highlight the need for interventions at multiple levels and scales,” commented co-author Prof Julia Arciero of Indiana University, USA.  

“While anti-inflammatory drugs are sometimes used to treat medical conditions associated with inflammation, we do not believe these are the whole answer for individuals,” said Dr David Katz, co-author and a specialist in preventive and lifestyle medicine based in the US. “Lifestyle changes such as healthy nutrition, exercise, and reducing exposure to stressful online content could also be important.”  

“The dawning new era of precision and personalized therapeutics could also offer enormous potential,” he added.  

At the societal level, the authors suggest creating calm public spaces and providing education on the norms and institutions that keep our societies stable and functioning.  

“While our ‘inflammation map’ hypothesis and corresponding mathematical model are a start, a coordinated and interdisciplinary research effort is needed to define interventions that would improve the lives of individuals and the resilience of communities to stress. We hope our article stimulates scientists around the world to take up this challenge,” Vodovotz concluded.  

The article is part of the Frontiers in Science multimedia article hub ‘A multiscale map of inflammatory stress’. The hub features a video, an explainer, a version of the article written for kids, and an editorial, viewpoints, and policy outlook from other eminent experts: Prof David Almeida (Penn State University, USA), Prof Pietro Ghezzi (University of Urbino Carlo Bo, Italy), and Dr Ioannis P Androulakis (Rutgers, The State University of New Jersey, USA). 

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