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Japan approves Novartis' Zolgensma for spinal muscular atrophy in patients under two years old

Published on 19/03/20 at 11:30am

Novartis has revealed that its gene therapy Zolgensma (onasemnogene abeparvovec) has secured approval from Japan’s Ministry of Health, Labour and Welfare (MHLW) for the treatment of spinal muscular atrophy (SMA) in the country in patients less than two years of age, including those who are pre-symptomatic at the time of diagnosis.

The decision was backed up by data drawn from Phase 1, 1/2 and 3 trials. These data showed that Novartis’ drug showed “rates of survival never seen in the natural history of the disease”, and led to improved achievement of disease-related milestones, including the ability to sit without support, a milestone “never achieved in untreated patients”.

In addition, the therapy showed rapid improvement in motor function in patients, often within the first month following dosing.

Zolgensma is a one-time treatment delivered via a single intravenous infusion, providing a working copy of the SMN1 gene, the absence of which is the defining factor in SMA. It is estimated that between 15 and 20 Japanese patients each year will be eligible to receive the treatment.

“SMA is the leading genetic cause of infant death and, if left untreated in its most common form, Type 1, leads to death or the need for permanent ventilation by the age of two in more than 90% of cases,” commented Kazunari Tsunaba, President and Representative Director at Novartis. “A one-time dose of Zolgensma has the potential to make a truly transformative impact on this life-threatening disease. This is an important day for the children and families in Japan impacted by SMA, both today and in the future.”

Matt Fellows

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