Sarepta, Biogen drugs with accelerated approval under fire in FDA report
A U.S. FDA report questions the agency's accelerated approval process, highlighting Sarepta's Exondys 51 and Biogen's Aduhelm as examples. The report criticizes the lack of thorough evaluations and missing analyses in the original plans for both drugs. Aduhelm was withdrawn by Biogen in 2024 due to poor sales, while Sarepta's drug remains under scrutiny. The report emphasizes the need for better oversight in the accelerated approval process, noting that 21 out of 24 drugs reviewed followed a consistent approach, with concerns focused on these three outliers.
A report from the U.S. FDA's Office of Inspector General is calling into question the thoroughness of the agency's accelerated approval process, and is using treatments from Sarepta Therapeutics (NASDAQ:SRPT) and Biogen (NASDAQ:BIIB) as examples.
The Sarepta and Biogen therapies mentioned are, respectively, Exondys 51 (eteplirsen) and Aduhelm (aducanumab). They are used for Duchenne muscular dystrophy and Alzheimer's disease.
The FDA's accelerated approval program allows treatment for which there is an unmet medical need to be approved faster than the traditional process. However, drugmakers whose treatments are granted accelerated approval are required to conduct postmarketing trials to confirm the drug's benefit; otherwise, the FDA could order it removed from the market.
Aduhelm was pulled from the market in 2024, though not at the request of the agency. Biogen, citing disappointing sales, made the decision, focusing resources on its newer Alzheimer's treatment, Leqembi (lecanemab). It also abandoned the required post-market study on Aduhelm.
The OIG report points out that for both eteplirsen and aducanumab, FDA evaluated analyses were not included in the sponsors' original analysis plans. Also, concerns about the efficacy of both drugs was brought up by both FDA scientist and advisory committee members.
OIG also noted that in the case of aducanumab, Biogen's meetings with the agency were not fully documented in the administrative files.
The report looked at 24 drugs that went through the accelerated approval process. It said that for 21 of them, "there was a consistent approach through the pathway," adding its concerns are focused on the three outlier drugs. Besides Exondys 51 and Aduhelm, the third drug is Makena, which was approved to reduce the risk of preterm birth, but was removed from the market in 2023 after a post-marketing study failed to confirm its efficacy.
"Given these outliers and the importance of FDA’s role in approving drugs for the public, it is critical that FDA have appropriate guardrails in place to offset risk and ensure consistent and appropriate practices," the report concludes. "FDA's primary guardrail, the confirmatory trial, has been limited in execution."