Context:

Sarepta Therapeutics is resisting the FDA's request to cease distribution of its gene therapy Elevidys, despite its association with three patient deaths due to liver failure. The FDA raised concerns after these incidents, urging a pause in shipments, especially since the treatment had been controversial due to limited evidence of its benefits. Elevidys, approved to slow the progression of Duchenne muscular dystrophy, faced criticism after failing to demonstrate significant improvements in patients' motor abilities in trials. While Sarepta plans to stop shipments to patients in advanced stages using wheelchairs, it continues distribution to ambulatory patients, maintaining no new safety issues have arisen in this group. The situation has intensified debates on FDA's drug approval leniency, and Sarepta's recent workforce cuts and executive promotions added complexities to its operational challenges, reflected by a significant drop in its stock price.

Dive Deeper:

• Sarepta Therapeutics is defying the FDA's request to halt shipments of Elevidys, a Duchenne muscular dystrophy treatment, after reports of patient deaths from liver failure, arguing there's no new safety risk for patients who are not wheelchair-bound.

• The FDA's request came amid concerns over the drug's approval, which was initially pushed through against some internal opposition, highlighting broader debates about the agency's approval standards, particularly under its accelerated approval program.

• Elevidys was meant to slow muscle degeneration in Duchenne patients, but its efficacy was questioned when late-stage trials failed to show significant motor ability improvements, though it increased a key protein production seen in clinical trials.

• Amidst safety concerns, Sarepta added a 'boxed warning' to Elevidys and reported a third death linked to an experimental therapy using similar technology, prompting the FDA to halt related studies.

• Sarepta's operational and financial distress is evident as it laid off a third of its workforce while not initially disclosing the third patient's death, contributing to a 36% drop in its stock value amid ongoing regulatory and public scrutiny.