Biogen’s SPINRAZA (nusinersen) has been approved by the FDA for the treatment of spinal muscular atrophy (SMA) in pediatric and adult patients.
 
The drug was under Priority Review and 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 approval was based on positive results from multiple clinical studies in more than 170 patients.  In ENDEAR, a pivotal controlled clinical study, patients treated with SPINRAZA achieved and sustained clinically meaningful improvement in motor function compared to untreated study participants. Also, a greater percent 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 and maintained milestones at ages when they would be expected to be lost. These findings support the effectiveness of SPINRAZA across the range of SMA patients and support the early initiation of treatment.