Today, the U.S. Food and Drug Administration (FDA) approved Zolgensma (onasemnogene abeparvovac-xioi), the first gene therapy for a neuromuscular disease. Zolgensma is a one-time intravenous (into the vein) infusion for the treatment of pediatric patients less than 2 years of age with spinal muscular atrophy (SMA) with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene, . . .
Today, Novartis, the parent company of AveXis Inc., announced that the U.S. Food and Drug Administration (FDA) has accepted the company’s Biologics License Application (BLA) under Priority Review for Zolgensma (formerly known as AVXS-101), a gene therapy for the treatment of spinal muscular atrophy (SMA) type 1. Priority Review status requires the FDA to review . . .