This week, AveXis, a Novartis company, reported that it has submitted a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) requesting approval of AVXS-101 to treat infants with spinal muscular atrophy (SMA) type 1. AveXis is also submitting regulatory applications for the therapy in Europe and Japan. If approved, AVXS-101 would be the first gene therapy for neuromuscular disease treatment.

AVXS-101 is a gene therapy that targets the root cause of SMA by delivering a gene to replace the missing or mutated survival of the motor neuron 1 (SMN1) gene. AveXis’ BLA is focused on data gathered from trials in which AVXS-101 was administered to infants with SMA type 1 through intravenous injection. Therefore, it is anticipated that potential FDA approval would initially be for infants with SMA type 1.

The FDA has 60 days to decide whether it will accept the BLA, which includes specific information about the drug, pre-clinical and clinical data, and manufacturing information. If the FDA accepts the application, the administration will then have six months to review it and make a decision on whether or not to approve AVXS-101.

The drug is still being tested in clinical trials of patients with various types of SMA, as well pre-symptomatic SMA patients. For some of these trials, enrollment is complete, and others are still recruiting patients. For information regarding eligibility criteria for currently enrolling or planned clinical trials, view the listings on www.ClinicalTrials.gov or www.studysmanow.com, or contact medinfo@avexis.com.