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# Gottlieb Believes CBER Will Probe Sarepta Approval
- URL: https://www.fdaweb.com/gottlieb-believes-cber-will-probe-sarepta-approval/
- Published: 2025-06-18T12:00:00.000Z
- Updated: 2026-09-14T15:14:41.000Z
- Author: David McFarland
- Tags: Biologics, #legacy-id-D5159384

Former FDA commissioner **Scott Gottlieb** expects FDA will reevaluate Sarepta’s approval of Duchenne muscular dystrophy (DMD) gene therapy Elevidys based on two recent patient deaths ([see story](https://www.fdaweb.com/elevidys-shipping-nixed-after-2nd-liver-failure-death/)) and the past controversial nature of former CBER head **Peter Marks** review interference in 2024 ([see earlier story](https://www.fdaweb.com/deja-vu-marks-overrides-reviewers-on-duchenne-drug-again/)). Marks overrode his then-Center’s review teams in expanding the limited approval of Elevidys to full approval. In granting accelerated approval in 2023, Marks had also interfered and wrote a [decisional memo](https://www.fda.gov/media/169707/download?ref=fdaweb.com) saying: “Although I agree with the review team’s conclusions regarding product quality and safety, I disagree with certain interpretations of the efficacy data and come to a different conclusion regarding individuals aged four through five years,” which cemented the product’s use.

Speaking on CNBC’s [*Squawk Box* 6/17](https://youtu.be/1%5Ff6P0yw7Q4?si=YetZWZhPGqRqgGH%5F&ref=fdaweb.com), Gottleib said he believes that new CBER director **Vinay Prasad** will closely examine the recent events and the approval in general because he has previously written about, and criticized Marks' role in the therapy’s approval. Gottlieb pointed out how Prasad is in an “unusual” role as someone who was politically appointed as CBER director. “He’s expressed interest in some of these past approvals, and I’ve heard he's also been taking a look at some of the pending CAR-T approvals," Gottlieb said. “I think what happens if the Center director starts getting involved in these regulatory decisions in sort of an arbitrary way then that creates unpredictability in the development space, and people don’t know where to allocate capital." 

Regarding Sarepta’s approval, Gottlieb said he did not see anything wrong with the way that went through the agency originally. “There are occasionally these exceptional cases where you do have Center directors taking an active interest in these new approvals that really establish new paradigms,” he said. “And the gene therapy approvals, the early ones were such products… But this has become a bugaboo, if you will. And a lot of people have argued that the agency in the past was too lax on some of these approvals, and too permissive when it came to these gene therapy technologies and other technologies as well. There’s sort of a handful of these cases that everyone seems to point to and rally around, and Vinay has done that with this approval in the past. He’s written about it and talked about it, so I don't expect him to step away from that past dialog.”