FDA Approves Spinraza for the Treatment of Spinal Muscular Atrophy

The FDA has approved Spinraza (nusinersen) for the treatment of spinal muscular atrophy (SMA) in adults and children. The drug can be administered into the fluid surrounding the spinal cord via injection.

The FDA’s Center for Drug Evaluation and Research helped determine the efficacy of Spinraza. A clinical trial looked at how Spinraza could help 121 patients with infantile-onset SMA who were diagnosed before 6 months of age and who were less than 7 months old at the time of their first dose.

During an interim analysis involving 82 of the 121 participants, 40% of patients who were treated with Spinraza achieved improvement in motor milestones defined in the study. None of the control patients showed similar results.

The most common adverse effects associated with Spinraza are upper respiratory infection, lower respiratory infection, and constipation. Low blood platelet count and toxicity to the kidneys are also possible.

"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,” Billy Dunn, MD, director of the Division of Neurology Products in the FDA's Center for Drug Evaluation and Research, said in a press release.

Biogen markets Spinraza, and Ionis Pharmaceuticals helped develop the drug.