Sarepta Therapeutics Faces 40% Stock Drop: What This Means for the Future of Gene Therapy

Shares of Sarepta Therapeutics tumbled over 30% recently, raising concerns about the future of its gene therapy treatment, Elevidys. The FDA is reportedly asking Sarepta to halt all shipments of this therapy. However, the company claims it hasn’t received any official direction from the FDA.

FDA Commissioner Marty Makary mentioned the agency is reviewing whether Elevidys should continue to be sold. This scrutiny comes after two patient deaths were linked to Elevidys, and a third death was reported involving a different experimental gene therapy from the same company.

Since its initial approval, Elevidys has stirred debate. While it was conditionally approved by the FDA for young patients with Duchenne Muscular Dystrophy (DMD)—a severe condition that weakens muscles—its actual effectiveness for these patients remains uncertain. DMD often leads to loss of mobility and can reduce life expectancy to the early 20s, highlighting the urgent need for effective treatments.

In 2023, Elevidys received conditional approval for children aged 4 to 5—the demographic that showed the most promise in clinical trials. By the next year, the FDA granted full approval for patients aged 4 and older who could still walk, and an accelerated approval for those who could not. However, this latter decision was controversial because evidence of its effectiveness in advanced cases was limited.

Adding to the complexity, Elevidys did not meet its objectives in a Phase 3 trial, but Sarepta contended it delivered encouraging results in other areas. Peter Marks, the former head of the FDA’s Center for Biologics Evaluation and Research, supported expanding Elevidys’s approval despite staff recommendations to the contrary.

Currently, Sarepta’s stock has plummeted by over 87% this year, a significant downturn that reflects the skepticism surrounding Elevidys. As discussions evolve, both patients and investors are anxious to see how regulatory decisions will unfold.

For further details on the FDA’s investigations, you can check Bloomberg’s coverage here.

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