Zolgensma (AVXS-101) Under Priority FDA Review as Possible Gene Therapy for SMA Type 1
The U.S. Food and Drug Administration (FDA) has granted priority review to Novartis’ Biological License Application (BLA) asking that Zolgensma (onasemnogene abeparvovec-xxxx) — previously called AVXS-101 — be approved as a gene therapy for spinal muscular atrophy (SMA) type 1. Its request is reported to specifically cover intravenous…