Novartis: FDA Priority Review of spinal muscular atrophy med

• The AVXS-101, now known as ZOLGENSMA^® (onasemnogene abeparvovec-xxxx)[1], filing is supported by data from the START trial which demonstrated a dramatic increase in survival and transformative improvement in achievement of developmental milestones compared to the natural history of SMA Type 1[2]
• SMA Type 1 is a progressive neuromuscular disease and the leading cause of genetic mortality in infants globally [3]
• ZOLGENSMA^® represents the first in a proprietary platform to treat rare, monogenic diseases using gene replacement therapy – technology that replaces a missing or defective gene with a functional copy to correct the underlying cause of genetic disease

Novartis today announced that the U.S. Food and Drug Administration (FDA) has accepted the company’s Biologics License Application (BLA) for AVXS-101, now known as ZOLGENSMA^® (onasemnogene abeparvovec-xxxx)[1], an investigational gene replacement therapy for the treatment of spinal muscular atrophy (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.[4] 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.[3] Left untreated, a baby’s muscles become progressively weaker eventually leading to paralysis or death, in most cases by his or her second birthday.[5] Delivered as a single, one-time infusion, this breakthrough 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.[2]

“This important step by the FDA brings us ever closer to delivering ZOLGENSMA to patients with SMA Type 1. Babies affected by this rare disease are currently faced with debilitating disease progression and lifelong invasive chronic treatment. As a one-time infusion that addresses the genetic root cause of SMA without the need for repeat dosing, ZOLGENSMA represents a potentially significant therapeutic advance for these patients and their families,” said David Lennon, president of AveXis. “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.”

https://www.marketscreener.com/NOVARTIS-9364983/news/Novartis-announces-FDA-filing-acceptance-and-Priority-Review-of-AVXS-101-a-one-time-treatment-des-27694301/