At the end of 2016, the American Food and Drug Administration (FDA) granted approval for the use of Spinraza (nusinsersen) for the treatment of spinal muscular atrophy (SMA) in paediatric and adult patients. This is unprecedented for the SMA community witnessing the first approved drug for this disease.
The FDA approval was based on positive results from multiple clinical studies in more than 170 patients. GOSH was the highest UK recruiter for the first study, ENDEAR, a Phase 3, controlled study evaluating Spinraza in infantile-onset, which took place in the BRC-supported Somers Clinical Research Facility.
The interim analysis of the data from ENDEAR, (previously reported in the December BRC newsletter) was considered throughout the approval process. In particular, patients with infantile-onset SMA recruited to the ENDEAR study and treated with Spinraza, achieved and sustained clinically meaningful improvement in motor function compared to untreated study participants. Furthermore, a greater percentage of patients on Spinraza survived compared to untreated patients. In open-label studies, some patients also achieved unexpected milestones such as ability to sit unassisted, stand or walk.