2023 new drug approvals in review: which rising star holds the most lucrative promise?
AI-translated from the Chinese original · editorially reviewed
In 2023 the U.S. FDA approved 55 new drugs — the second-highest total in history — with breakthroughs in gene therapy, respiratory syncytial virus, Alzheimer's disease, and more. Pfizer emerged as the big winner with seven approvals, while the new drug forecast to hold the most commercial promise is Leqembi, the Alzheimer's drug developed by Biogen and Eisai.
New drug approvals surge, and gene therapy comes into its own
The U.S. FDA approved 55 new drugs (small molecules and biologics) in 2023, up 50% from 37 in 2022 and the second-highest total on record, behind only the 59 approvals of 2018. The gap between 2023 and 2022 likely reflects pandemic-related delays in the approval process.
By therapeutic area, oncology again led the field with the most new approvals (13 drugs, 24% of the total). Neurological diseases such as Alzheimer's and migraine ranked second (9 drugs, 16%), with infectious diseases and hematologic conditions tied for third (5 drugs each, 9%). Notably, 28 of the 55 newly approved drugs (51%) were orphan drugs for rare diseases, offering new options for patients in urgent need of new treatments.
The FDA approved a wide range of gene therapies, vaccines, and blood products this year, with the most eye-catching being the expensively priced gene therapies: Casgevy, the first gene-editing therapy based on CRISPR–Cas9 (priced at US$2.2 million); Lyfgenia, approved for the same indication (sickle cell disease, priced at US$3.1 million); Roctavian for adults with severe hemophilia A (priced at US$2.9 million); and Elevidys for Duchenne muscular dystrophy (priced at US$3.2 million). Duchenne muscular dystrophy is a rare disease caused by an abnormality on the X chromosome — a missing segment of the DMD gene — that leaves the muscle fiber membrane weak and fragile and leads to muscle wasting.
The first RSV vaccines arrive, and Alzheimer's sees a new breakthrough
Another major story of 2023 was the approval of respiratory syncytial virus (RSV) vaccines. GSK led the way in May with Arexvy for adults aged 60 and over, followed by Pfizer's Abrysvo, approved for pregnant women as well as adults 60 and over. In July, Beyfortus, the antibody developed by Sanofi and AZ, was approved for preventing RSV infection in infants. Sales of these products are projected to reach US$1.3–1.7 billion by 2028.
As for the closely watched Alzheimer's field, Leqembi, developed by Biogen and Eisai, is the first drug shown in clinical trials to effectively clear amyloid plaques from the brain, and the first anti-beta-amyloid antibody in years to receive traditional full approval, bringing new hope to Alzheimer's patients. Leqembi's rival, Eli Lilly's investigational antibody donanemab, also posted positive Phase 3 data, and the FDA was expected to rule on its application for full approval in early 2024.
Which company won the year, and which rising star holds the most lucrative promise?
Pfizer took the crown in 2023 with the most approvals (seven), more than double any other company — the last time a single company racked up so many was in 2019, when Novartis earned six.
Pfizer's newly approved drugs include the multiple myeloma therapy Elrexfio; Ngenla, a growth hormone developed with OPKO; Litfulo, a first-in-class treatment for severe alopecia areata; the RSV vaccine Abrysvo; and the COVID-19 antiviral Paxlovid, which received full approval. Its remaining two approvals came via 2022 acquisitions: the ulcerative colitis drug Velsipity (from its US$6.7 billion acquisition of Arena) and the nasal-spray migraine drug Zavzpret (from its US$11.6 billion acquisition of Biohaven).
Among the other contenders, GSK, Biogen, Chiesi, and UCB each earned three approvals. GSK's were the myelofibrosis drug Ojjaara, the kidney disease drug Jesduvroq, and the RSV vaccine Arexvy. Biogen's were the ALS drug Qalsody, the Alzheimer's drug Leqembi co-developed with Eisai, and the postpartum depression drug Zurzuvae co-developed with Sage. Chiesi's were Fulsuvez for a rare skin disease and two enzyme replacement therapies, Lamzede and Elfabrio. UCB won approval for Bimzelx for plaque psoriasis and the myasthenia gravis drugs Zilbrysq and Rystiggo.
AZ's RSV antibody Beyfortus and the Alzheimer's therapy Leqembi may be the most commercially promising of 2023's approvals — Beyfortus is projected to reach peak sales of US$1.9 billion, while Leqembi, buoyed by the vast Alzheimer's patient population, could reach peak sales of US$4.8 billion.
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