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.
