Posted by Michael Wonder on 03 Dec 2018
Novartis announces FDA filing acceptance and priority review of AVXS-101, a one-time treatment designed to address the genetic root cause of SMA Type 1
3 December 2018 - The AVXS-101, now known as Zolgensma (onasemnogene abeparvovec-xxxx), filing is supported by data from the START trial which demonstrated a dramatic increase in survival and transformative improvement in achievement of developmental milestones compared to the natural history of SMA Type 1.
Novartis today announced that the U.S. FDA has accepted the company's biologics license application for AVXS-101, now known as Zolgensma (onasemnogene abeparvovec-xxxx), an investigational gene replacement therapy for the treatment of spinal muscular atrophy (SMA) Type 1. Zolgensma is designed to address the genetic root cause of SMA Type 1, a deadly neuromuscular disease with limited treatment options.
Zolgensma previously received breakthrough therapy designation and has been granted priority review by the FDA, with regulatory action anticipated in May 2019.
