AVXS 101 filed in the EU, US and Japan for spinal muscular atrophy.- AveXis.

AveXis announced it has submitted regulatory applications for AVXS 101 (gene therapy) in the U.S., Europe and Japan for use in infants with type 1 Spinal Muscular Atrophy (SMA). Data from the pivotal Phase I study conducted in SMA Type 1 formed the primary basis for these submissions.

Based on the data included in the applications, it is hoped that the initial label will be for intravenous (IV) use of AVXS 101 for infants with SMA Type 1, as IV dosing has only been studied in clinical trials in infants. The study of AVXS 101 in SMA Type 2 (STRONG) is ongoing, and data from that study will help determine the final study design for the planned study in children up to 18 years of age (REACH). Novartis is hoping to launch AVXS 101 in the middle of 2019.