Novartis drug improves SMA treatment in kids: 6 European study findings

Spine

The Novartis gene therapy drug Zolgensma has led to significant improvements in motor function for presymptomatic children with spinal muscular atrophy, European Pharmaceutical Review reported June 21.

Six notes:

1. SMA is a rare genetic disease that leads to progressive muscle weakness and, in some cases, paralysis or death.

2.  Zolgensma is the only gene therapy for SMA and the only SMA treatment designed to directly address the genetic root cause of the disease. The drug replaces the defective or missing SMN1 gene to halt disease progression with a single infusion, administered to patients over one hour.

3. In the Spr1nt phase 3 clinical trial, all children treated presymptomatically were independent of respiratory and nutritional support and met the primary endpoint of sitting independently for 30 seconds or more.

4. Seventy-nine percent of children who achieved the sitting milestone did so within the World Health Organization window of normal development, according to the report.

5. In the Str1ve trial, 82 percent of children achieved developmental motor milestones not observed in the natural history of SMA Type 1, including 49 percent who sat without support for 30 seconds or more.

6. The results of the trials were hailed as "extraordinary" by Shephard Mpofu, MD, senior vice president and CMO at Novartis Gene Therapies. "When treated with Zolgensma prior to the onset of symptoms, not only did all patients survive, but were thriving — breathing and eating on their own and sitting independently, with many standing and walking," Dr. Mpofu said.

Click here for more details on the studies.

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