FDA restricts Sarepta’s gene therapy Elevidys after Duchenne patient deaths

The FDA has imposed new restrictions on Sarepta Therapeutics’ gene therapy, Elevidys, following reports of fatalities among patients with Duchenne muscular dystrophy (DMD). The updated label now features a boxed warning and introduces a new “limitation of use,” specifically excluding non-ambulatory patients from receiving the treatment. This regulatory action underscores the agency’s heightened scrutiny of gene therapies, particularly in vulnerable patient populations.

This decision comes in the wake of serious safety concerns that have emerged since Elevidys’ approval. The implications for Sarepta are significant, as the company must now navigate a more complex regulatory landscape while addressing the safety concerns that have arisen. For pharma professionals in regulatory, QA/QC, and CMC roles, this situation highlights the critical importance of robust risk management strategies and the need for ongoing vigilance in monitoring patient outcomes in gene therapy trials.