AveXis Announces Alignment with FDA on Next Steps Toward a BLA Submission
for AVXS-101 in SMA Type 1
Company to submit information requested by FDA to the IND on an on-going basis
AveXis plans to request a pre-BLA meeting in Q2 2018
Conference call and webcast today at 4:30 pm EST
Chicago, Ill. (January 4, 2018) AveXis, Inc. (NASDAQ: AVXS), a clinical-stage gene therapy company developing treatments for patients suffering from rare and life-threatening neurological genetic diseases, today provided an update following the receipt of minutes from the end-of-Phase 1 meeting with the U.S. Food and Drug Administration (FDA) conducted on December 5, 2017, regarding the companys primary gene therapy candidate, AVXS-101, for the treatment of spinal muscular atrophy (SMA) Type 1.
The goal of the end-of-Phase 1 meeting was to review the non-clinical, clinical and Chemistry, Manufacturing and Controls (CMC) data that has been generated by AveXis to date, and to align with the FDA on next steps leading to a Biologics License Application (BLA) submission.
The FDA provided detailed information requests in each of the areas discussed, which the company plans to address by submitting the requested information to the investigational new drug (IND) application on an on-going basis. AveXis has been working on many of these areas of focus in anticipation of the requests at some point during the review process. AveXis also plans to provide available data from its on-going pivotal trial of AVXS-101 in SMA Type 1 (STR1VE) prior to the pre-BLA meeting.