Sarepta Defies FDA Order Amidst Gene Therapy Controversies

Sarepta Therapeutics has taken an unusual step by declining the FDA's request to cease shipments of its gene therapy after a third patient death associated with the treatment. The decision highlights ongoing controversies surrounding the company's practices and regulatory compliance.

The FDA, emphasizing the need for immediate action where serious safety signals are detected, is at odds with Sarepta's stance. Although the FDA can pull drugs from the market, this process is typically prolonged, and companies generally agree to informal suspension requests.

This development comes as Sarepta's stock has suffered significantly, compounded by recent layoffs. The gene therapy Elevidys, initially approved amidst skepticism, remains under scrutiny, particularly concerning its safety and efficacy for patients with Duchenne's muscular dystrophy.