FDA approves Spinraza (nusinersen) for the treatment of spinal muscular atrophy (SMA) in pediatric and adult patients.- Biogen

The FDA has approved Biogen’s Spinraza (nusinersen) under Priority Review for the treatment of spinal muscular atrophy (SMA) in pediatric and adult patients. Spinraza is the first and only treatment approved in the U.S. for SMA, a leading genetic cause of death in infants and toddlers that is marked by progressive, debilitating muscle weakness.

The FDA approval of Spinraza was based on positive results from multiple clinical studies in more than 170 patients. The data package included the interim analysis of ENDEAR, a Phase III controlled study evaluating Spinraza in infantile-onset, as well as open-label data in pre-symptomatic and symptomatic patients with, or likely to develop, Types 1, 2 and 3 SMA.

In the ENDEAR study, a pivotal controlled clinical study, infantile-onset SMA patients treated with Spinraza achieved and sustained clinically meaningful improvement in motor function compared to untreated study participants. In addition, a greater percentage of patients on Spinraza survived compared to untreated patients. In open-label studies, some patients achieved milestones such as ability to sit unassisted, stand or walk when they would otherwise be unexpected to do so and maintained milestones at ages when they would be expected to be lost. The overall findings of these studies support the effectiveness of Spinraza across the range of SMA patients, and appear to support the early initiation of treatment.