Convaid | R82 community partners Cure SMA will hold a webinar updating the community on the status of Spinraza, the first-ever approved therapy for Spinal Muscular Atrophy. The states in purple are already administering the drug. Cure SMA will continue to update the site list as they confirm more sites that are administering or preparing to administer Spinraza.
The Spinraza webinar will be held on Thursday, May 4, at 1PM EDT (10AM PDT/11AM MDT/12PM CDT). Click here to register.
According to the Cure SMA website, on December 23, the FDA announced that it has approved SpinrazaTM (nusinersen) to treat spinal muscular atrophy, making it the first-ever FDA-approved therapy for SMA.
Cure SMA writes, “We are thrilled to see our community’s efforts culminate in the approval of Spinraza: not only the first-ever approved treatment for this disease, but also one that addresses the underlying genetic cause of SMA. This has been a story of all groups—families, researchers, companies and the FDA—working together as one community to reach this amazing milestone. We are especially pleased that the sophisticated and rigorous clinical development plan that Biogen and Ionis chose to implement has resulted in a broad label that will now give so many patients access.The approval from the FDA for all SMA—pediatric and adult—is the broadest possible label, with no restrictions—and this matches our core value at Cure SMA of being one united community for all ages and all types of SMA.”
Convaid | R82 recently joined Cure SMA in Orange County walk-n-roll event where Biogen executives were on hand to discuss roll-out of their groundbreaking drug. Convaid | R82 have long supported awareness building of Spinal Muscular Atrophy and of the work done by Cure SMA.
“Biogen is committed to continuing to work together with the SMA community as we embark on a future where there is now a treatment available for this devastating disease,” said George A. Scangos, PhD, chief executive officer at Biogen. “The teams at Biogen and Ionis are grateful for the support we have received and we join Cure SMA and SMA families in celebrating this critical milestone for the community.”
“There has been a long-standing need for a treatment for spinal muscular atrophy, the most common genetic cause of death in infants, and a disease that can affect people at any stage of life,” said Billy Dunn, MD, director of the Division of Neurology Products in the FDA’s Center for Drug Evaluation and Research. “As shown by our suggestion to the sponsor to analyze the results of the study earlier than planned, the FDA is committed to assisting with the development and approval of safe and effective drugs for rare diseases and we worked hard to review this application quickly; we could not be more pleased to have the first approved treatment for this debilitating disease.”