Dyne Therapeutics files FDA BLA for DYNE‑251 in Duchenne muscular dystrophy, supported by DELIVER trial data and Priority Review request.
Written By: Fariha Sameen, PharmD
Reviewed By: Pharmacally Editorial Team
Dyne Therapeutics has submitted a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) seeking accelerated approval of zeleciment rostudirsen (DYNE‑251) for individuals with Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping. The filing covers a 20 mg/kg once‑monthly regimen and is supported by positive data from the Phase 1/2 DELIVER trial (NCT05524883).
Dyne has also requested Priority Review, which, if granted, could shorten the FDA’s review timeline from 10 months to six months following the agency’s 60‑day filing review. On this schedule, a potential U.S. launch could occur in the first quarter of 2027.
DELIVER Trial Supports Filing
The BLA submission is based on results from the registrational expansion cohort of the global Phase 1/2 DELIVER trial, which met its primary endpoint. Dyne reported clinically meaningful functional improvement alongside a favorable safety and tolerability profile. The therapy continues to be evaluated in the long‑term extension phase of DELIVER and in the confirmatory global Phase 3 FORZETTO study, which is intended to support full approval.
Advancing Beyond Current Therapies
Existing exon-skipping therapies such as eteplirsen, golodirsen, and viltolarsen have expanded treatment options for DMD but still require frequent dosing and deliver variable dystrophin restoration. Dyne’s antibody-conjugated PMO platform aims to improve tissue delivery and support monthly dosing, addressing ongoing unmet need in the DMD community
John Cox, president and chief executive officer of Dyne, emphasized the company’s focus on advancing efficacy, safety, and dosing convenience for patients with this progressive disease.
Expanding Duchenne Pipeline
Beyond DYNE‑251, Dyne is advancing additional exon‑skipping candidates targeting exons 53, 45, 44, and 55 through programs DYNE‑253, DYNE‑245, DYNE‑244, and DYNE‑255. Together, these programs aim to broaden therapeutic reach across multiple DMD genotypes.
Global Development Pathway
Zeleciment rostudirsen has received Breakthrough Therapy, Fast Track, Rare Pediatric Disease, and Orphan Drug designations from regulatory agencies in the U.S., Europe, and Japan. These designations support Dyne’s accelerated global development strategy, though formal submissions outside the U.S. have not yet been detailed.
Targeting Dystrophin Restoration
DMD is a rare, X‑linked neuromuscular disorder caused by mutations in the DMD gene, resulting in severe dystrophin deficiency and progressive muscle degeneration. Exon‑skipping therapies aim to restore functional dystrophin by bypassing faulty genetic sequences during RNA processing.
DYNE‑251 combines a phosphorodiamidate morpholino oligomer (PMO) with a transferrin receptor 1‑targeting antibody fragment to enhance delivery into muscle tissue and the central nervous system. The approach is designed to increase near‑full‑length dystrophin production while reducing treatment burden through monthly dosing.
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About the Writer
Fariha Sameen, PharmD (LinkedIn), is a clinical pharmacy professional with hands-on experience in patient counselling, medication review, therapeutic monitoring, and clinical documentation across multiple departments. She has experience identifying and assessing drug-related problems and supporting medication safety practices. Her interests include pharmacovigilance, ADR reporting, clinical research, and medical writing focused on clear, evidence-based communication.