A Phase 3 Multinational, Randomized, Double-Blind, Placebo-Controlled Systemic Gene Delivery Study to Evaluate the Safety and Efficacy of SRP-9001 in Subject With Duchenne Muscular Dystrophy (EMBARK)

Sarepta Therapeutics, Inc. has conducted multiple studies for the treatment of Duchenne Muscular Dystrophy (DMD). The EMBARK study is their first for gene therapy for DMD. The study is focuses on DMD patients ages 4-5 years of age. The study’s purpose is to evaluate the safety and efficacy of gene transfer therapy administer via intravenous infusion. The study is conducted over a two-year period. Study participants are assigned to receive study drug at either year one or year two and receive placebo the other year. The study is conducted in a double-blind matter where participants and research staff are not aware of the assignment.

In June 2023, the FDA granted Accelerated Approval for boys with DMD ages 4-5. This achievement marks a milestone as the first FDA approved gene-therapy for DMD. The study visits continue to collect long-term safety and efficacy data. Studies are planned for different populations of DMD boys in hopes to open the accessibility of the drug to all ages in the future.