FDA Adcomm Tentatively Recommends Sarepta’s Duchenne Muscular Dystrophy Gene Therapy

An advisory committee for the US Food and Drug Administration (FDA) has voted 8 to 6 to recommend accelerated approval for Sarepta Therapeutics Duchenne muscular dystrophy gene therapy. The narrow vote comes despite concerns from several regulators about the treatment’s benefit-risk profile and data supporting the application.

According to Max Bayer, “Sarepta argued that a surrogate endpoint quantifying the expression of micro-dystrophin protein in patients’ muscles was an adequate biomarker to predict clinical benefit and grant accelerated approval. The company cited a natural history study as evidence that correcting the expression addresses the root cause of DMD. The FDA has for years been wary of the proposed biomarker, suggesting as far back as 2018 that Sarepta reconsider the endpoint. ”

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(Source: Fierce Biotech, May 12^th, 2023)