Lack of Transparency Tarnishes Sarepta’s Sheen as Patient Deaths Trigger FDA Battle

Three recent patient deaths have been linked to Sarepta Therapeutics' gene therapy products, including Elevidys for Duchenne muscular dystrophy and an investigational therapy for LGMD25.

Sarepta's failure to promptly disclose information about the most recent patient death has drawn criticism from investors, Wall Street analysts, and the FDA, raising serious concerns over transparency15.

The FDA has responded by pausing all clinical trials for LGMD gene therapies and revoking Sarepta’s platform designation for its viral vector technology—the same platform used for both Elevidys and the LGMD therapy5.

The FDA has also asked Sarepta to halt all shipments of Elevidys in the U.S., but the company has initially refused, stating it will continue shipments for ambulatory (walking) patients15.

This standoff with the FDA is highly unusual and has contributed to a sharp decline in Sarepta’s share price—over 35%—and led to increased risk that Elevidys could be withdrawn from the market entirely23.

The controversy comes at a time of broader challenges in the gene therapy sector, including regulatory scrutiny, investment withdrawal, and heightened safety concerns13.

Sources:

1. https://www.biopharmadive.com/news/sarepta-fda-elevidys-duchenne-gene-therapy-crisis-questions/753515/

2. https://www.genengnews.com/topics/genome-editing/stockwatch-sarepta-shares-nosedive-after-lgmd-gene-therapy-patient-dies/

3. https://www.fiercepharma.com/pharma/fda-mulls-elevidys-market-withdrawal-following-3rd-death-after-sarepta-gene-therapy

5. https://www.biospace.com/fda/lack-of-transparency-tarnishes-sareptas-sheen-as-patient-deaths-trigger-fda-battle