AveXis is working with the U.S. Food and Drug Administration (FDA) to overcome final regulatory hurdles before starting a pivotal trial of AVXS-101 for the treatment of spinal muscular atrophy type 1 (SMA type 1) later in 2017.
AVXS-101 is a gene therapy for SMA that has shown great promise in the treatment SMA type 1 in an ongoing Phase 1 trial (NCT02122952).
The company announced last week that its Good Manufacturing Practice (GMP) commercial manufacturing process for AVXS-101 aligns with FDA requirements laid out in the recently released minutes of a Type B Chemistry Manufacturing and Controls (CMC) meeting with the agency.
A Phase 1/2a trial of AVXS-101 for treating SMA type 2 is also projected to start later this year.
For AveXis to proceed with the planned clinical trials, the FDA must accept the method to be used to assess the potency assay qualification of AVXS-101, a virus-based therapy that must be carefully calibrated through biological testing.
The FDA acknowledged the potency assay proposed by AveXis has the potential to meet its requirements, but that more information was needed to predict the feasibility of the plans. AveXis is working to address this request for more information and to have the data ready to submit to the FDA in August.
AveXis now has its own manufacturing facility for AVXS-101, where it has produced the product for the planned pivotal trials using the new manufacturing process. Use of the product awaits FDA acceptance of the potency assay qualification. It also awaits FDA agreement that batches of the product are appropriate for a Phase 3 study.
An end-of-Phase 1 meeting with the FDA is projected for August. This is when data on the degree of similarity between the AVXS-101 product used in the clinical trial of SMA type 1 and the product made according to the new manufacturing process (the comparability protocol), along with the potency assay qualification to be included with the data on the full Phase 1 clinical trial, will be reviewed and discussed. Regulatory options are also likely to be reviewed.
“The goal of the CMC meeting was to align with FDA on our commercial manufacturing process, analytical methods, and comparability protocol, all three of which we believe were achieved in this collaborative and constructive discussion,” Sean Nolan, president and CEO of AveXis, said in a press release.
“The team has already made progress toward addressing the FDA’s request regarding potency assay qualification, and we anticipate only a modest impact to timelines,” he said.
“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,” Nolan said.