FDA Lets AveXis Know the Information It Needs to Possibly Approve AVXS-101 for SMA Type 1

The U.S. Food and Drug Administration has given AveXis a list of information it needs to consider approving AVXS-101 as a treatment for spinal muscular atrophy Type 1.

Company officials met with regulators to discuss the results of a Phase 1 clinical trial (NCT02122952) of the gene therapy. They also discussed what AveXis should include in the Biologics License Application it plans to file for the treatment. This kind of application covers biological as opposed to pharmaceutical treatments.

The Phase 1 study is one of a number of trials that AveXis has conducted, or is conducting, of the therapy’s ability to treat different types of SMA.

“We are very pleased that the constructive and collaborative discussion during the end-of-Phase 1 meeting resulted in the identification of the specific next steps we must take on our path to a BLA [Biologics License Application] submission for AVXS-101 in SMA Type 1,” Sean Nolan, president and chief executive officer of AveXis, said in a press release.

“We greatly appreciate the level of clarity we received from the FDA and will provide our responses,” Nolan said. AveXis hopes to “request a pre-BLA meeting in the second quarter of 2018,” he added.

At that meeting the FDA will let AveXis know about any additional information it needs in the application.

The company said it will provide regulators with the results of its ongoing Phase 3 STR1VE trial (NCT03306277) of AVXS-101 before the meeting. The trial in infants with SMA type 1 is expected to run until 2020.

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Findings of another Phase 1 trial prompted FDA officials to say that they believe the therapy has the potential to prevent the progression of SMA.

The agency earlier granted AVXS-101 orphan drug status for all types of SMA. It also gave the treatment breakthrough therapy status and fast track status for SMA Type 1. Those designations are aimed at accelerating the drug’s regulatory approval for the condition.

In addition, AveXis is studying AVXS-101 in a Phase 1 trial (NCT03381729) called STRONG as a treatment for children with SMA type 2.

AVXS-101 is composed of a harmless virus that delivers a functional SMN gene — which is faulty in SMA — to nerve cells that control movement. The goal is to prevent the cells from degenerating.

In related news, AveXis and Regenxbio announced an expansion of a licensing agreement, initiated in 2014, for the development and commercialization of SMA treatments. Under the new agreement, AveXis receives exclusive rights to all vectors in Regenxbio’s NAV technology platform for use in potential SMA therapies in exchange for upfront and other payments, a press release states.

AVXS-101 uses the Regenxbio’s AAV9 vector — a non-replicating, harmless virus called an adeno-associated virus — to deliver the corrected gene to SMA patients.