On November 14, 2025, the U.S.
Food and Drug Administration (FDA) made significant updates to the label of Sarepta Therapeutics’ gene therapy, Elevidys, specifically designed for Duchenne muscular dystrophy (DMD).
This pivotal update includes crucial changes such as the introduction of a boxed safety warning and a clear restriction that the therapy is intended solely for use in ambulatory patients.
These updates stem from ongoing concerns regarding the treatment’s safety and effectiveness, underscoring the FDA’s commitment to ensuring that patients receive therapies that are both safe and beneficial.
Moreover, the FDA has mandated that a comprehensive postmarketing study be conducted, aiming to monitor the long-term safety and efficacy of Elevidys within its approved patient population.
This article will delve deeper into the FDA’s recent label updates, their implications for patients, and the anticipated future studies surrounding gene therapy for Duchenne muscular dystrophy.
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