As Sarepta Therapeutics waits for the Food and Drug Administration to announce an approval decision on a new drug to treat Duchenne muscular dystrophy, the biotech is years behind schedule on a requirement to confirm the muscle-function benefit of its first and top-selling medicine Exondys 51.
The FDA has until Monday to decide if Sarepta can market a second drug called Vyondys 53 to treat patients, mostly young boys, with the inherited and fatal muscle-wasting disease. Approval would accelerate Sarepta’s revenue growth because it targets a different genetic form of Duchenne than Exondys 51.
But the long-term commercial success of both drugs is contingent on Sarepta producing definitive scientific proof of a clinical benefit for Duchenne patients. For Exondys 51, whose generic name is eteplirsen, that requirement is taking longer than expected — raising questions about Sarepta’s effectiveness and its commitment to fully meet FDA-mandated obligations.
This article is exclusive to STAT+ subscribers
Unlock this article — plus daily coverage and analysis of the biotech sector — by subscribing to STAT+.
Already have an account? Log in
Already have an account? Log in
To submit a correction request, please visit our Contact Us page.
STAT encourages you to share your voice. We welcome your commentary, criticism, and expertise on our subscriber-only platform, STAT+ Connect