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# FDA Mulls Pulling Sarepta’s Gene Therapy
- URL: https://www.fdaweb.com/fda-mulls-pulling-sareptas-gene-therapy/
- Published: 2025-07-18T12:00:00.000Z
- Updated: 2026-09-14T15:17:02.000Z
- Author: David McFarland
- Tags: Biologics, #legacy-id-D5159535

An HHS official tells ***FDA Webview*** the agency is taking a "hard look" at pulling from the market Sarepta Therapeutics’ Elevidys (delandistrogene moxeparvovec-rokl), an adeno-associated virus vector-based gene therapy for treating Duchenne muscular dystrophy patients. The move is in response to two reports of fatal acute liver failure following treatment of non-ambulatory pediatric male Duchenne patients with the gene therapy ([see earlier story](https://www.fdaweb.com/safety-communication-on-2-elevidys-related-deaths/)). In the meantime, the agency reportedly will ask Sarepta to voluntarily stop all Elevidys shipments until its regulatory evaluation is complete.

Earlier on Friday, the company disclosed the [death of another patient](https://www.reuters.com/business/healthcare-pharmaceuticals/sarepta-shares-slump-after-third-patient-death-this-year-2025-07-18/?ref=fdaweb.com) who received a separate Sarepta experimental gene therapy, expanding product safety concern over use of its treatments. A 51-year-old man enrolled in an early-stage trial of gene therapy SRP-9004 for treating limb-girdle muscular dystrophy had succumbed to acute liver failure.

Last month, former FDA commissioner **Scott Gottlieb** predicted that the agency would reevaluate Elevidys’ approval based on the two recent patient deaths 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 by 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 believed that new CBER director **Vinay Prasad** will closely examine the recent events and the approval in general because he had 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.”