Scrutiny Intensified: FDA Calls Halt on Sarepta's Gene Therapy Amid Safety Concerns

The Food and Drug Administration (FDA) has asked Sarepta Therapeutics to voluntarily halt shipments of its Elevidys gene therapy. The request came following the death of a muscular dystrophy patient who was receiving a different, experimental treatment, raising safety alarms about similar gene technologies.

Sarepta, based in Cambridge, has refused the request for ambulatory patients, citing no new safety concerns in this group. However, they continue a shipment halt for non-ambulatory patients, as initiated on June 15 after an acute liver failure incident. This aligns with a broader FDA suspension on trials for limb girdle muscular dystrophy.

Wall Street reacted sharply, with Sarepta's shares dropping significantly amid heightened scrutiny. Patient advocacy groups express concern over the implications for Duchenne muscular dystrophy. Sarepta has agreed to add a liver toxicity warning to their Elevidys packaging, amid ongoing investigations into safety risks.