Prime Highlights:

• Sarepta will stop U.S. deliveries of Elevidys after FDA action based on grounds of safety.
• Human being treated, the death due to liver failure has been the source of concern.

Key Facts:

• There are three deaths attributed to Sarepta’s gene therapy platform.
• The company is also facing job loss and program cuts on grounds of financial pressures.

Key Background

Sarepta Therapeutics initiated a short-term hold on Elevidys shipments throughout the United States last week. It is at the behest of the U.S. Food and Drug Administration (FDA) that this action has been initiated after two cases of acute liver failure were reported in adolescent patients receiving treatment for Elevidys, a gene therapy to treat Duchenne muscular dystrophy (DMD). The therapy, which is a product of a proprietary adeno-associated viral (AAV) vector, has been under investigation because of its possible safety issues, mainly relating to liver toxicity.

This suspension will take full effect by Tuesday evening, although the company has indicated that patients who are already under ambulatory treatment on current indications will be allowed to proceed. The halt is generally reserved for new patients and non-ambulatory patients, a sign of increased regulatory oversight as Sarepta and the FDA move to quell fear with revised safety labeling.

The break happened as increasing fiscal stress is imposed upon Sarepta. Its shares fell over 8% after hours, and the company had already witnessed a sharp 40% share price free-fall in the last week or so. As a way of reducing costs, Sarepta has embarked on a sweeping corporate transformation that includes laying off about 500 workers and shelving some gene therapy development projects. The measures are intended to redirect funds to high-priority projects and use the company’s money better.

Elevidys had received accelerated FDA approval in June 2023, initially in limited form for ambulatory DMD patients in the range of 4 to 5 years old. Sarepta has continued with its efforts since then to expand its market, however, even as it too has been increasingly under heavy fire for the riskiness of the treatment. Recent deaths and regulatory action point to ongoing issues with gene therapy, specifically a balance problem between therapeutic breakthrough potential and patient safety. Sarepta’s future plans are to work closely with the FDA and potentially resume distributing Elevidys upon the addition of mutual safety improvements.