FDA Panel Votes on Gene Transfer Therapy for Duchenne Muscular Dystrophy

FDA Panel Votes on Gene Transfer Therapy for Duchenne Muscular Dystrophy

The Food and Drug Administration’s (FDA) Cellular, Tissue and Gene Therapies Advisory Committee voted 8 to 6 in support of accelerated approval of delandistrogene moxeparvovec (SRP-9001) for the treatment of ambulatory patients with Duchenne muscular dystrophy (DMD) with a confirmed mutation in the DMD gene.

Delandistrogene moxeparvovec is an investigational gene transfer therapy designed to deliver a shortened, functional component of dystrophin to muscle tissue. Under the accelerated approval program, the FDA can approve a treatment earlier based on a surrogate endpoint thought to predict clinical benefit, which in this case would be the expression of SRP-9001 dystrophin protein.