Biogen Communicates Updated NURTURE Study Results for the European Community
24 October 2019
The NURTURE study is an ongoing Phase 2, open-label study of pre-symptomatic patients with SMA, who received their first dosing before six weeks of age. Nusinersen was administered to 25 infants six weeks old or younger, who were in the pre-symptomatic stage, genetically-diagnosed with SMA and likely to clinically develop Type 1 or 2.
The following updated results were formally presented at the Cure SMA Annual SMA Conference in Anaheim, California and the 5th Congress of the European Academy of Neurology (EAN) in Oslo, Norway.
All study participants were alive, without a need for permanent ventilation and 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. Overall, the study showed that nusinersen was well-tolerated with no safety concerns identified after up to nearly four years of treatment.
As the NURTURE study continues, these interim results add to the longest study of SMA pre-symptomatic infants. After up to 45.1 months of analysis, nusinersen continues to demonstrate efficacy and safety in comparison to the natural history of SMA. The study median age was nearly three years old, with 88 percent of the study participants walking independently, with many of them doing so in the normal timeframe for an infant. As the community will be aware, in the natural history of SMA, patients with SMA Type 1 or Type 2 are never able to walk independently. This new data shows that patients treated with nusinersen had continuous improvement in sitting and walking independently, with the overwhelming majority of patients achieving motor milestones in a normal timeframe.