FDA Scrutiny Intensifies as Sarepta Gene Therapy Faces Dire Setbacks

The FDA is set to request Sarepta Therapeutics to halt shipments of its gene therapy, Elevidys, after a third patient's death. This marks a heightened scrutiny following previous deaths linked to the therapy. Sarepta's shares plummeted amid concerns over management transparency and safety warnings.

The U.S. Food and Drug Administration is poised to request Sarepta Therapeutics to cease deliveries of its gene therapy Elevidys, according to insiders. The company's shares fell 36% to $14.08 after an over 40% drop during trading, following the announcement of a third patient's death connected to its gene therapies.

A spokesperson for Sarepta mentioned the absence of an official request from the FDA thus far. Increased scrutiny follows the deaths of two adolescents earlier in the year who had received Elevidys, a therapy aimed at treating Duchenne muscular dystrophy. The FDA's concerns mounted after these events, despite earlier patient support and the former head of gene therapy, Peter Marks, granting approval.

Sarepta revealed that Friday marked the death of a third patient, a 51-year-old with limb-girdle muscular dystrophy, who suffered acute liver failure. The news raises questions over the therapy's safety, already highlighted by the use of adeno-associated virus vectors. Analysts predict mounting patient hesitancy, and the company is facing challenges regarding disclosure practices and management credibility.

(With inputs from agencies.)