AveXis Submits SMA Gene Therapy for Regulatory Approval in U.S., Europe, and Japan

Rare Daily Staff

AveXis, in a letter addressed to the spinal muscular atrophy community, said it submitted regulatory applications in the United States, Europe, and Japan, for use of its SMA type 1 gene therapy AVXS-101 in infants.

SMA is a severe neuromuscular disease characterized by the loss of motor neurons leading to progressive muscle weakness and paralysis. It is caused by a genetic defect in the SMN1 gene that codes SMN, a protein necessary for survival of motor neurons. The most severe form of SMA is type 1, a lethal genetic disorder characterized by motor neuron loss and associated muscle deterioration, which results in mortality or the need for permanent ventilation support before the age of two for greater than 90 percent of patients.

“This is an important and exciting initial step toward the potential approval of AVXS-101,” the company said.

Data from the company’s pivotal phase 1 study conducted in SMA type 1 formed the primary basis for its submissions. Based on the data included in the applications, the company said it expected that the initial label will be for intravenous use of AVXS-101 for infants with SMA type 1, as IV dosing has only been studied in clinical trials in infants.

The company said the U.S. Food and Drug Administration will make the decision of whether to accept its application for AVXS-101 within the next 60 days. If accepted, the company anticipates the FDA will take six months to review the application and decide on whether to approve AVXS-101. That means the company could have a potential approval for the gene therapy in the first half of 2019

The company said the European Medicines Agency could act on the application in mid-2019 and Japan in mid-September.

October 18, 2018