Novartis’s Biologics License Application (BLA) for Zolgensma, designed to tackle the genetic root cause of spinal muscular atrophy (SMA) Type 1, a deadly neuromuscular disease, was accepted by the U.S. Food and Drug Administration (FDA).
AVXS-101 (Zolgensma), an investigational gene replacement therapy for the treatment of this disease with limited treatment options. Zolgensma earlier got Breakthrough Therapy designation and has been granted Priority Review by the FDA, which is planned to be regulated further, in May 2019.
SMA is caused by a defective or missing SMN1 gene, and without a functional SMN1 gene, infants with SMA Type 1 rapidly lose the motor neurons responsible for muscle functions such as breathing, swallowing, speaking and walking. If left untreated, a baby’s muscles become progressively weaker eventually leading to paralysis or death, in most cases by his or her second birthday.
David Lennon, president of AveXis, a Novartis company, that works on novel treatments for patients suffering from rare and deadly neurological genetic diseases, said that a one-time infusion that addresses the genetic root cause of SMA removes the need for repeat dosing. “The introduction of one-time, potentially curative therapies will require rethinking how our healthcare system manages diagnosis, treatment, care and associated costs for patients with genetic disease. Novartis and AveXis are proud to lead the way toward a modern healthcare system built on the tremendous value of truly innovative and transformative medicines that could bend the curve of life. We are committed to flexibly partnering with healthcare stakeholders to ensure appropriate access to our medicines,” Lennon said.
Approval in Japan and Europe in 2019
In Japan and Europe, where Zolgensma has SAKIGAKE and PRIME (PRIority MEdicines) designations, comparable to FDA’s Breakthrough Therapy designation, it is expected to get a decision by regulators in first half and mid of 2019.