FDA approval numbers soar after COVID troubles

Market Access
approval numbers

The FDA released its overview of 2023 and the drugs it had approved during the year. Ben Hargreaves finds that the agency’s approval numbers have bounced back, after being substantially down in 2022, and that 2023 could be remembered due to some landmark approvals.

In recent history, the US Food and Drug Administration (FDA) has been charting a course that saw the number of novel drug approvals steadily increasing over the years. This trend was disrupted by 2022, where approvals plunged to the lowest number in six years, as well as falling 26% compared to the year prior. At 37 approvals in 2022, this represented a major drop from the record year in 2018, where 59 were given the go-ahead.

However, in 2023, there was a return to higher figures, as the FDA announced that 55 new drugs were approved in the US. This figure was some way above the average number of approvals over the last decade, suggesting that normal business is being resumed. Among those treatments given the nod, there were some medicines, such as gene therapies, that could make 2023 a landmark year for approvals.

A blip

The reasons why 2022’s figures were so low is not entirely clear, but clues were provided in a statement at the end of the year by Patrizia Cavazzoni, director at the Center for Drug Evaluation and Research. In an end-of-year review, Cavazzoni made immediate reference to the impact that COVID-19 had taken on the global community, and also referenced the emergence of Mpox during the year. “Despite these hardships, we approved many therapies to prevent, diagnose, and treat a wide range of diseases and conditions,” Cavazzoni stated.

Similarly, research conducted on 2022’s approvals found that the slowdown in approvals was not likely to be due to the industry itself, but more likely due to an administrative slowdown. “If we were to assess the strength of the pharmaceutical industry in 2022 based on the number of new drugs accepted by the FDA, the conclusion could be worrying, as the number has been significantly lower than in previous years (37 vs. an average of 52.5 for the last 4 years). However, it should be considered that the process of putting a drug onto the market is slow and that this number may therefore reflect only a slowdown in bureaucratic and administrative processes,” the authors state.

Another factor that may have influenced the lower than average rate of approvals could be related to the external pressures placed on the FDA by the accelerated approval of Aduhelm (aducanumab). The FDA’s decision on the drug came against the recommendation of its advisory committee, where ten out of 11 members voted against the treatment receiving accelerated approval. The FDA’s interaction with Biogen during the review process was pulled apart in a US congressional report, which labelled communication between the two as “inappropriate” and “atypical”. Accelerated approval was granted to Aduhelm mid-way through 2021, and immediately proved controversial. Though perhaps unrelated, the following year saw 19 drugs being rejected by the FDA, compared to just three in 2023.

A return to previous highs

Regardless of the reasons for low approval rates in 2022, the subsequent year saw levels return to previous highs. As previously mentioned, the 55 drugs approved is some way higher than the recent average, and significantly higher than the preceding decade, which averaged approximately 46 approvals per year. The higher rate of accepting marketing applications is part of a trend that has occurred over the last decade, with rates of approval on a steady upward trajectory that reached an all-time high in 2018, when 59 drug applications were approved.

Among the treatments approved in 2023, there were some standout medicines that represent firsts for the industry. Notably among these was Eisai and Biogen’s Leqembi (lecanemab), the Alzheimer’s disease therapy that became the second approval in the space after the partner’s controversial previous treatment, Aduhelm. The therapy is the first to be able to show an ability to slow the cognitive decline in early Alzheimer’s patients.

The FDA also provided approvals to gene therapies, which included bluebird’s Lyfgenia (lovotibeglogene autotemcel), and CRISPR Therapeutics and Vertex Pharma’s Casgevy (exagamglogene autotemcel). Both treatments are approved to treat sickle cell disease in patients 12 years or older, but Casgevy is notable as being the first-ever approved therapy that utilises CRISPR gene editing. The treatment is created in such a way that it is able to ‘cut’ DNA in targeted areas, allowing it to remove, add, or replace DNA. It is one of a growing ‘one-and-done’ gene therapies, which only require one treatment and the patient is potentially cured.

Outside of these major approvals, there was also an approval for Eli Lilly’s Zepbound (tirzepatide) as a treatment for obesity. The therapy joins Novo Nordisk’s Wegovy (semaglutide) as a part of an emerging wave of weight loss treatments, which is becoming a major focus across the industry, with their efficacy and commercial potential becoming more evident.

Across the pond

With the FDA’s number of approvals rising, it is useful to look to the European Medicines Agency (EMA) to see whether the European agency had a similar rise in figures. Historically, the FDA and EMA approved a similar number of drugs, with the EMA also noting in 2019 that it is aligned with the FDA on more than 90% of its marketing authorisation decisions.

However, in 2023, the EMA’s figures on its positive opinions on new medicines had fallen, in comparison to 2022 numbers. In 2023, 77 total positive opinions were given, while 89 were provided in 2022. The number also compares unfavourably with 2021 and 2020 figures, 92 and 97, respectively. Since 2015, this is the second-lowest number of positive opinions, behind only the 66 authorisations in 2019. Though on the lower side, there have been multiple occasions of positive opinions in the low 80s during the last decade, meaning it is not drastically different from previous years.

With the disruption of the pandemic years, both the EMA and the FDA’s figures will be better understood in the years to come, whether as an exception or as part of a larger trend. However, ahead in 2024, there is another significant year of potential approvals that could arrive. According to a Clarivate report, this year will feature a number of drugs that could be set for approval that use new treatment modalities, and treatments for previously-untreatable or under-treated diseases. 

“The year 2024 is shaping up to be the best of times and the worst of times for the pharma industry,” stated Henry Levy, president of Life Sciences & Healthcare at Clarivate. “New modalities, underpinned by scientific breakthroughs in the past decade, are achieving clinical successes and providing treatments for patients with previously unmet medical needs. Meanwhile, externalities such as government initiatives to contain healthcare costs, the sustained high cost of capital, and global geopolitical disputes are putting a brake on investor appetites for the sector.”