Biologics

Dyne Therapeutics BLA for Duchenne Muscular Dystrophy

FDA has accepted for priority review a Dyne Therapeutics BLA for zeleciment rostudirsen for treating individuals with Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping. The company says it has requested accelerated approval based on dystrophin as a surrogate endpoint. The agency set a user fee review action target date of 1/21/2027.

The submission is based on data from the Phase 1/2 DELIVER clinical trial where z-rostudirsen met its primary endpoint. Dyne says treatment with z-rostudirsen once every four weeks resulted in a robust and statistically significant increase in dystrophin production with functional improvement observed across multiple clinical endpoints and a favorable safety profile. The company says Z-rostudirsen continues to be evaluated in the long-term extension portion of the DELIVER trial and in the global confirmatory Phase 3 FORZETTO clinical trial.

Z-rostudirsen comprises a phosphorodiamidate morpholino oligomer conjugated to an antigen-binding fragment that binds to the transferrin receptor 1, according to Dyne. It is designed to enable the production of near-full-length dystrophin in muscle and the central nervous system to provide functional improvement.

DMD is a rare X-linked progressive neuromuscular disorder caused by mutations in the DMD gene. These mutations result in a complete or near-complete absence of dystrophin, a protein critical for maintaining muscle structure and function. Symptoms typically appear between ages 3 and 5 and include progressive muscle weakness, loss of lower and upper limb function and eventually cardiac and respiratory failure, according to the company.

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Gupta Raises Concerns About Overton’s Independence, Ideology

Overton Confirmation Remains Uncertain After Senate Hearing

FDA Classifies Boston Scientific Imager II Catheter Recall as Class I

FDA Approves Gazyva for Childhood-Onset Idiopathic Nephrotic Syndrome