FDA grants Breakthrough Therapy Designation to AveXis’ (AVXS) AVXS-101 Gene Therapy for Pediatric Spinal Muscular Atrophy Type 1
AveXis, Inc. (NASDAQ: AVXS), a clinical-stage gene therapy biotech company focused on developing gene therapy for patients with rare and life-threatening neurological genetic diseases, announced on Wednesday, July 20, 2016, that the U.S. Food and Drug Administration has granted Breakthrough Therapy Designation to AVXS-101, its proprietary gene therapy candidate for the treatment of pediatric spinal muscular atrophy (SMA) Type 1. AVXS-101 had previously been granted orphan drug designation for the treatment of all types of spinal muscular atrophy (SMA) and Fast Track designation for the treatment of SMA Type 1. Breakthrough Therapy Designation is given to a drug intended alone or…