At the CureSMA conference this year Biogen on 1st July 2019 published new results from the NURTURE study.
In the NURTURE study, SPINRAZA was administered to infants six weeks old or younger (n=25), who were in the pre-symptomatic stage, genetically-diagnosed with SMA and had two or three copies of the SMN2 gene (n=15 for two copies (most likely to develop Type 1 SMA); n=10 for three copies (most likely to develop Type 2 SMA)).
These results add data to the longest study of spinal muscular atrophy (SMA) in pre-symptomatic infants (n=25). These data reported, after up to 45.1 months of analysis, continue to demonstrate efficacy and safety in patients treated pre-symptomatically with SPINRAZA in comparison to the natural history of this disease. These new data also showed that patients treated with SPINRAZA had continuous improvement with the overwhelming majority of patients achieving motor milestones in a normal timeframe.
These study results demonstrate the durable impact of pre-symptomatic, proactive treatment on transforming the natural course of this disease. We are seeing an extensive number of patients continually meeting child motor development milestones and making unprecedented gains in a previously hopeless and often fatal condition,” said Darryl De Vivo, M.D., Sidney Carter Professor of Neurology and Pediatrics, Columbia University Irving Medical Center in New York, New York. “SPINRAZA is setting patients on a path toward survival, greater mobility and independence, which is helping improve outcomes for patients of all ages.”
Results from NURTURE, an ongoing, Phase 2, open-label study of 25 pre-symptomatic patients with SMA (most likely to develop SMA Type 1 or 2) who received their first dose of SPINRAZA before six weeks old, demonstrated unparalleled results in comparison to the natural history of SMA. As of March 2019:
- 100 percent were alive without a need for permanent ventilation.
- The median age of the study participants was nearly three years old. The majority of untreated patients with SMA Type 1 never reach their second birthday without permanent ventilation.
- 100 percent of the infants were sitting independently, in comparison to the natural history of this disease where no patients with SMA Type 1 would be able to do so and patients with SMA Type 2 would need assistance.
- 88 percent of the infants were walking independently with many of them doing so in the normal timeframe for a toddler. In the natural history of SMA, patients with SMA Type 1 or Type 2 are never able to walk independently.
- Patients were approaching the maximum mean score of 64 on the CHOP INTEND measure of motor function– 63.4 for patients with 3 SMN 2 copies (n=10) and 62.1 for those with 2 SMN 2 copies (n=15), demonstrating the powerful impact of early SPINRAZA treatment.
- SPINRAZA demonstrated longer term efficacy up to nearly 4 years, with participants continuing to make progress and showing no signs of loss of motor function.
- SPINRAZA was well-tolerated with no new safety concerns identified after up to nearly 4 years of treatment.
Click here to view the full announcement.
The TreatSMA Team