Sarepta Stands Behind Elevidys After FDA Requests Withdrawal Amid Safety Concerns

The FDA requested Sarepta Therapeutics to suspend distribution of Elevidys, its gene therapy for Duchenne muscular dystrophy, after at least three patient deaths and rising safety concerns4.

The deaths, reportedly due to acute liver failure, occurred in patients treated with Elevidys or other gene therapies using the same AAVrh74 vector4.

Sarepta refused the FDA's voluntary request to halt all shipments of Elevidys, instead choosing to stand by the therapy45.

The FDA has also placed clinical holds on Sarepta's ongoing gene therapy studies for limb girdle muscular dystrophy and revoked the company's platform technology designation45.

The latest fatality reportedly occurred in June, but the FDA only took action in July after news became public; Sarepta had already faced criticism for its handling and delayed disclosure of safety events3.

Analysts see a rising likelihood the FDA will impose a formal market withdrawal, a move that could divide the patient community and has already caused Sarepta's stock to lose over 35% of value13.

Sources:

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

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

4. https://www.fda.gov/news-events/press-announcements/fda-requests-sarepta-therapeutics-suspend-distribution-elevidys-and-places-clinical-trials-hold

5. https://www.fiercebiotech.com/biotech/sarepta-ldmd-trials-all-hit-fda-hold-amid-newly-surfaced-safety-concerns