(RTTNews) - Dyne Therapeutics Inc. (DYNE), a clinical-stage company, on Monday announced the acceptance by the U.S. Food and Drug Administration (FDA) of the biologics license application (BLA) for zeleciment rostudirsen in treating Duchenne muscular dystrophy.
Duchenne Muscular Dystrophy (DMD) is a rare, X-linked genetic disorder that causes progressive muscle loss due to mutations in the DMD gene that controls dystrophin production.
Patients are often diagnosed at childhood and face a lifetime of mobility issues as muscle tissue gradually seizes, losing contraction and expansion abilities. There are approximately 12,000 individuals with DMD in the European Union and the U.S. alone.
Zeleciment rostudirsen (z-rostudirsen) is an investigational therapy for DMD patients with mutations in the DMD gene amenable to exon 51 skipping. The drug consists of a phosphorodiamidate morpholino oligomer (PMO) conjugated to an antigen-binding fragment (Fab) binding to the transferrin receptor 1 (TfR1). It is designed to enable the production of near-full length dystrophin in muscles and the central nervous system (CNS).
The therapy previously received an Orphan Drug designation by the FDA, Europen Medicines Agency (EMA), and Ministry of Health, Labour, and Welfare (MHLW) in Japan, and was additionally granted a Rare Pediatric Disease Voucher, Breakthrough Therapy, and Fast Track status from the FDA.
The BLA was supported by data from the Phase 1/2 DELIVER trial, which met its primary endpoint. The extension period of the trial is ongoing, yet the results gained so far have been submitted with the BLA, which will undergo a Priority Review and may facilitate an Accelerated Approval.
The global, confirmatory Phase 3 FORZETTA trial will continue to evaluate z-rostudirsen.
The FDA has set a prescription drug user fee act (PDUFA) date of January 21, 2027, by which time a response to the BLA will be issued. If approved, the company plans to launch the drug by the end of the first quarter in 2027.
DYN closed Friday at $23.74, up 4.49%. In the pre-market, shares are trading at $24.83, up 4.60%.
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