Incident and Impact
A patient died of acute liver injury, a known side effect of ' gene therapy , marking the first such death among over 800 patients treated. The case's severity was unprecedented.
Following the announcement, Sarepta's shares plummeted more than 23%, reflecting investor concern over the therapy's safety and the potential impact on its market value.
Regulatory and Financial Aspects
Elevidys, the first U.S. gene therapy for Duchenne muscular dystrophy, received expedited approval in 2023, despite some internal skepticism about its effectiveness. Full approval was granted last year for patients with a specific genetic mutation.
The therapy, costing $3.2 million for a one-time treatment, has been under scrutiny, with having secured accelerated FDA approval for three other Duchenne drugs since 2016, none of which have yet confirmed efficacy.