AveXis Announces Plan to Initiate Pivotal Trial of AVXS-101 in SMA Type 1 Using Product from New GMP Commercial Process
FDA notified AveXis it may initiate pivotal trial based on a review of data provided by the company following CMC Type B meeting
Product used in the pivotal trial will be from the AveXis GMP manufacturing process; pivotal trial to commence immediately
Conference call and webcast today at 8:00 a.m. EDT
Chicago, Ill. (September 29, 2017) AveXis, Inc. (NASDAQ: AVXS), a clinical-stage gene therapy company developing treatments for patients suffering from rare and life-threatening neurological genetic diseases, today announced the U.S. Food and Drug Administration (FDA) has notified the company that based on review of data submitted, including the potency assay, it may initiate its planned pivotal trial of AVXS-101 for patients with spinal muscular atrophy (SMA) Type 1 using the intravenous (IV) formulation produced by the companys Good Manufacturing Practice (GMP) commercial manufacturing process. The company
plans to initiate this trial immediately.
We are pleased to reach this outcome following a thorough review by the FDA of the voluminous information we supplied to address the commitments made during the Chemistry, Manufacturing, and Controls Type B meeting in May, and are eager to initiate our pivotal trial of AVXS-101 in SMA Type 1 in the U.S. using product from our GMP process, said Sean Nolan, President and Chief Executive Officer of AveXis. Moving AVXS-101 back into the clinic, as planned, with product from our GMP process is a significant milestone, not only for AveXis but also for the patients we hope to serve.