FDA approves first drug for spinal muscular atrophy

Spinraza improved motor function in SMA patients.

In ENDEAR, 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.

“Spinraza offers new hope for the SMA community and exemplifies our mission of applying cutting-edge science to make a meaningful difference in the lives of patients with devastating, life-altering diseases,” said George A. Scangos, CEO at Biogen. “We are humbled and grateful for the commitment of the patients and families who participated in the Spinraza clinical trial program, the tireless efforts of our investigators, and the urgency demonstrated by the FDA in rapidly reviewing and approving this treatment. We also want to acknowledge the important work of our colleagues at Ionis, who initiated this program.”

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 3 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.

“With the approval today of Spinraza, the future for those affected with SMA has changed. We are especially pleased that this sophisticated and rigorous clinical development plan has resulted in a broad label that may offer access to many patients,” said Kenneth Hobby, president at Cure SMA. “This has been a story of all groups – families, researchers, companies and the FDA – working together as one community.”

Spinraza will be made available for shipment in the US to healthcare providers in approximately one week. Biogen anticipates there may be variation in time to treatment as institutions and treatment centres learn about Spinraza.