Novartis: New SMART Study Data Continues To Support Zolgensma's Clinical Benefits In SMA

(RTTNews) - Novartis Pharma AG, affiliated to Swiss drug major Novartis AG (NVS), Monday presented new data from the SMART study that continue to support the clinical benefits of Zolgensma (onasemnogene abeparvovec), the only one-time gene therapy for the treatment of spinal muscular atrophy or SMA.

The SMART study supplements a growing body of evidence on the use of Zolgensma in a patient population older and heavier, i.e., 1.5 - 9.1 years of age, than the children treated in previous clinical studies.

According to the company, nearly all patients treated maintained or improved motor milestones after 52 weeks, with most switching to the one-time gene therapy from another chronically administered disease-modifying therapy.

SMA is a rare, genetic neuromuscular disease and a leading genetic cause of infant death, caused by the lack of a functional SMN1 gene. It is the most severe forms of SMA result in the rapid and irreversible loss of motor neurons, affecting muscle functions including breathing, swallowing and basic movement.

Zolgensma is the only approved gene therapy for the treatment of SMA and the only SMA treatment designed to directly address the genetic root cause of the disease. It replaces the function of the missing or non-working SMN1 gene to halt disease progression through sustained SMN protein expression with a single, one-time IV infusion.

The SMART study is the first open-label clinical study of Zolgensma to include previously treated patients.

Novartis said the final data from the SMART study highlight the safety and efficacy profile of Zolgensma in children with SMA, ranging in age from ~18 months to 9 years.

Most patients maintained or improved motor milestones from baseline at the time of final analysis. In the trial, nearly all patients who could sit with slight support still met this milestone at Week 52.

Three patients achieved the milestone of newly standing with support and one achieved newly walking with support at Week 52. All patients who could walk at baseline maintained this milestone until end of study.

Zolgensma is now approved in more than 51 countries and more than 3,700 patients have been treated with the drug worldwide across clinical trials, managed access programs, and in the commercial setting.

Novartis has an exclusive, worldwide license with Nationwide Children's Hospital to both the intravenous and intrathecal delivery of AAV9 gene therapy for the treatment of all types of SMA.

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