The Readout Loud 281: Decoding biotech hype, the Sarepta saga, & au revoir to a CEO
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Nov 2, 2023 Biotech consultant Frank David explains subgroup analyses and how they can be misleading. The podcast also discusses the future of Sarepta and the retirement of a biotech stalwart.
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Sarepta's DMD Trial Results
- Sarepta's Duchenne muscular dystrophy gene therapy trial technically failed its primary endpoint.
- However, subgroup analysis showed positive effects in younger boys (4-5 years old), creating a complex situation for FDA approval.
Debate Over Sarepta's Data
- Sarepta's trial results sparked debate: the biotech community points to flaws in the data and subgroup analyses.
- Families of affected boys and the company advocate for approval due to observed positive effects, creating an ethical dilemma.
Sarepta's History with the FDA
- Sarepta has a history of conditional approvals without successfully completing confirmatory studies.
- The FDA's leniency and flexibility in handling these situations is a recurring concern, similar to Alzheimer's drug approvals.