2023: A record year for approvals

Over the past ten years, the FDA’s novel drug green lights have averaged at about 46 per year. With 55 novel drug approvals, 2023 saw the highest tally of nods since 2018 and became the year with the greatest number of approvals in 30 years.

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Although the agency did not provide any explanation for the large drop in approvals in 2022, the huge gap between that year and 2023 could be attributed to the inspection delays arising from the COVID-19 pandemic. “The number of novel drugs approved varies from year to year, and may be due to a variety of factors,” the FDA said in a statement.

 

Boost for Rare Diseases

More than half, or 28 out of 55, of the novel drugs approved in 2023 fell into the sphere of rare diseases, including drugs for amyotrophic lateral sclerosis (ALS), Friedreich’s ataxia; paroxysmal nocturnal haemoglobinuria; and the genetic disorder known as activated phosphoinositide 3-kinase delta.  And, among 2023’s 16 cancer-related approvals, numbered therapies for rare cancers such as mantle cell lymphoma, and nasopharyngeal carcinoma.

“Our commitment to the rare disease community continued in 2023,” said Patrizia Cavazzoni, director of the CDER, a commitment that could already be seen in 2022, a year in which 54 percent of the new approvals were for drugs to treat rare diseases. Moreover, the push for rare disease drugs has been bolstered by regulatory incentives to encourage rare disease drug development, such as the Fast Track and Breakthrough Therapy designations.

 

Gene Therapy Breakthroughs

Gene therapies have also picked up momentum with the milestone approvals of two treatments for sickle cell disease – Casgevy from CRISPR, the first drug to reach the market using the firm’s novel gene editing technology, and Lyfgenia from Vertex. In addition, a hemophilia A therapy from BioMarin along with one for Duchenne muscular dystrophy from Sarepta also succeeded.

According to Tim Hunt, CEO of the Alliance for Regenerative Medicine (ARM), speaking at the Biotech Showcase 2024 investor conference, these approvals represent a “seminal moment in the history of biotechnology.”

Despite the achievement these approvals represent in the field, the large price tag for gene therapies – USD 2.2 million per patient for Casgevy for example – raises the question of how the US healthcare system could support these costs.

 

Big Winners

Pfizer was last year’s top approvals performer. Perhaps a product of the acquisitions the company undertook with its massive COVID-19 profits, Pfizer scored a total of seven novel drug green lights. Pfizer drugs approved include Velsipity for the treatment of moderate to severe ulcerative colitis, and the nasal spray Zavzpret for migraine, acquired from its purchase of Biohaven.

Other Pfizer products to achieve FDA recognition were its Paxlovid COVID-19 antiviral, which gained a full approval; the multiple myeloma drug Elrexfio; its first-in-class alopecia treatment Litfulo, and its arexvy respiratory syncytial virus (RSV) vaccine Abryvso.

But Pfizer was not the only big pharma to earn valuable nods in 2023. Eli Lilly saw its blockbuster obesity treatment, Zepbound, authorised for chronic weight management, while Eisai and Biogen garnered approval for their Alzheimer’s treatment Leqembi.

GSK also reaped a significant go-ahead for its RSV vaccine in addition to the Pfizer antibody for the same indication, both for adults, while Sanofi and AstraZeneca got a nod for their vaccine, Beyfortus, which protects infants.

Full list of approvals:

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