(RTTNews.com) - Swiss drug major Novartis AG ( NVS ) announced Monday that the U.S. Food and Drug Administration has accepted the company's Biologics License Application or BLA for AVXS-101, now known as ZOLGENSMA (onasemnogene abeparvovec- xxxx), an investigational gene replacement therapy for the treatment of spinal muscular atrophy or SMA Type 1.
ZOLGENSMA is designed to address the genetic root cause of SMA Type 1, a deadly neuromuscular disease with limited treatment options. ZOLGENSMA previously received Breakthrough Therapy designation and has been granted Priority Review by the FDA, with regulatory action anticipated in May 2019.
SMA is caused by a defective or missing SMN1 gene. 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. Left untreated, a baby's muscles become progressively weaker eventually leading to paralysis or death, in most cases by his or her second birthday.
Delivered as a single, one-time infusion, the technology works by replacing the missing or defective SMN1 gene with a functional copy that makes SMN protein, thereby improving motor neuron function and survival.
In Japan, where ZOLGENSMA has SAKIGAKE Designation, a decision by regulators on the New Drug Application or J-NDA is expected in the first half of 2019. In Europe, where ZOLGENSMA has PRIME (PRIority MEdicines) designation, a decision by regulators on the Marketing Authorization Application (MAA) is expected in mid-2019.
The SAKIGAKE and PRIME designations are comparable to the FDA's Breakthrough Therapy designation. These regulatory applications are based primarily on data from the START trial.
Priority Review designation means the FDA's goal is to take action on an application within six months, compared to 10 months under standard review.
Read the original article on RTTNews (http://www.rttnews.com/2959388/novartis-fda-accepted-bla-for-sma-type-1-gene-replacement-therapy-zolgensma.aspx)
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