AveXis has aligned with the FDA regarding the compliance of its manufacturing process for gene therapy AVXS-101 with Good Manufacturing Practice (GMP). This benchmarks FDA support for the company’s manufacturing route, carried out at their own facility, for the commercial manufacture of the proprietary candidate. The initial product made at the facility is planned for use in two upcoming trials of AVXS-101 in patients with varying types of Spinal Muscular Atrophy (SMA).
The final remaining caveat from the FDA is the full implementation of its potency assay qualification plan, prior to the initiation of upcoming clinical studies. Currently, the FDA has commented that not enough information regarding these has been provided to assess the feasibility of the plans. The relevant data is expected to be ready by August this year.
AVXS-101 is a gene therapy that targets the underlying monogenic cause of SMA, and is effective in crossing the blood-brain barrier, thus facilitating an intravenous route of delivery and effective targeting.
CEO Sean Nolan commented that the meeting was successful in cooperating with the FDA on ‘commercial manufacturing process, analytical methods and comparability protocol, all three of which we believe were achieved in this collaborative and constructive discussion.’ He further stated, ‘We are pleased with the outcomes of the meeting and the progress we have made at the AveXis facility, and, most importantly, believe we have a scalable GMP commercial process in place to fulfill future patient demand and a path forward to potentially utilize the Phase 1 data in our regulatory pathway.’
Source: AveXis Announces Alignment with FDA on GMP Commercial Manufacturing Process for AVXS-101 Press Release