Today, Dyne Therapeutics announced that the U.S. Food and Drug Administration (FDA) has accepted the company’s Biologics License Application (BLA) for zeleciment rostudirsen (z-rostudirsen) for the treatment of individuals living with Duchenne muscular dystrophy who are amenable to exon 51 skipping. The application has been accepted for Priority Review, and the FDA has assigned a Prescription Drug User Fee Act (PDUFA) target action date of January 21, 2027 for potential accelerated approval based on dystrophin expression as a surrogate endpoint.
Z-rostudirsen is an investigational exon skipping therapy bound to an antigen-binding fragment (Fab) to help the therapy reach the muscle cells for those amenable to exon 51 skipping. The BLA is supported by data from the Phase 1/2 DELIVER clinical trial, which demonstrated increased dystrophin production along with improvements across multiple functional endpoints and a favorable safety profile.
What This Means
The FDA’s acceptance of the application means the agency has determined the submission is complete and will now begin its formal review to determine whether the therapy should be approved for people living with Duchenne who are amenable to exon 51 skipping. Priority Review shortens the FDA’s review timeline from the standard 10 months to approximately six months for applications that may offer significant improvements in treatment.
An FDA acceptance of a BLA is not an approval. During the review period, the FDA will evaluate the therapy’s safety, effectiveness, manufacturing, and overall benefit-risk profile before making a final decision.
PPMD is pleased to learn this news and will continue to monitor the review process and share updates with the Duchenne community as they become available.
Read Dyne’s press release here.
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