Sarepta Resumes Supply of Gene Therapy After FDA Review

• The FDA recommended resuming supply for young patients
• Sarepta's shares rose by over 16% following the announcement of the resumption
• Elevidys is the first approved therapy for muscular dystrophy

Washington — Shares of Sarepta Therapeutics surged by over 16% following the announcement of the resumption of supply of gene therapy for certain patients. This decision was made after a temporary pause requested by regulators.

The FDA recommended lifting the restrictions for young patients with a diagnosis of Duchenne muscular dystrophy, who can still walk. The pause was initiated after the death of two adolescents who underwent treatment, but the FDA clarified that a recent report of the death of an 8-year-old boy was not related to the therapy.

This gene therapy, known as Elevidys, is the first approved in the U.S. for treating Duchenne muscular dystrophy, a life-threatening condition that primarily affects boys and young men. The therapy received accelerated approval in 2023 for a limited cohort of young patients and is set to be expanded in 2024 for use in older patients, including those who cannot walk.

Analysts believe that the FDA's decision significantly improves the sales prospects for Elevidys in the near term. However, the FDA noted that the company must provide new data on safety for the resumption of therapy for older patients.

The FDA will continue to work with the company regarding patients who cannot walk, which remains a prerequisite for voluntary pause following two deaths.

Tags: USA