Rare-Disease Evidentiary Crackdowns: Sarepta's Elevidys and Replimune's RP1

Summary

Two rare-disease decisions defined the combative core of Dr. Vinay Prasad’s FDA tenure. In July 2025, Prasad led the FDA to request that Sarepta Therapeutics suspend all shipments of Elevidys, its Duchenne muscular dystrophy gene therapy priced at $3.2 million per dose, after patient deaths; the agency also placed Sarepta’s limb-girdle muscular dystrophy gene-therapy trials on clinical hold. The halt was later lifted for ambulatory patients after a third attributed death was determined to be unrelated. The episode triggered an intense backlash from industry and patient groups and preceded Prasad’s brief first resignation — he was reinstated within two weeks.

Separately, the FDA denied Replimune’s RP1 melanoma therapy twice — a July 2025 complete response letter and an April 2026 denial — demanding a randomized control arm to isolate RP1’s contribution to efficacy. In April 2026, a Wall Street Journal opinion piece blamed Prasad by name for the denial. The Replimune fight was still unresolved when Prasad departed the FDA at the end of April 2026.

Prasad had argued before joining the agency that Elevidys’s approval was a prime example of regulators bending evidence standards for industry: in a 2024 letter co-authored with Timothée Olivier, he said former CBER director Peter Marks “overruled 3 review teams and pushed this product on the market despite a failed trial.” His FDA actions put that criticism into enforcement practice.

Timeline

• July 2025 — FDA, under Prasad, requests Sarepta suspend all Elevidys shipments after patient deaths; LGMD gene-therapy trials placed on clinical hold.
• Late July 2025 — Prasad resigns amid backlash, then is reinstated within two weeks; the Elevidys halt is lifted for ambulatory patients after a third attributed death is found unrelated.
• July 2025 — FDA issues a complete response letter denying Replimune’s RP1 melanoma therapy, demanding a randomized control arm.
• April 2026 — FDA denies RP1 a second time; a Wall Street Journal op-ed blames Prasad by name.
• April 30, 2026 — Prasad departs the FDA; the RP1 dispute remains unresolved.