FDA urges Elevidys to pause after third death; Sarepta refuses

CAMBRIDGE, Massachusetts: A third patient death linked to gene therapy technology has intensified scrutiny on Sarepta Therapeutics, whose Duchenne muscular dystrophy treatment, Elevidys, remains at the centre of safety concerns.

Despite a request from U.S. regulators to halt shipments, the company is refusing to pause deliveries for ambulatory patients.

On July 18, the Food and Drug Administration confirmed that it had asked Sarepta to voluntarily stop distributing Elevidys, a gene therapy for Duchenne muscular dystrophy. The request came during a meeting between the agency and the company.

Sarepta declined the FDA’s request, stating it would continue shipping Elevidys to patients who are able to walk while maintaining an existing pause for non-ambulatory patients — a…