Drugs

Ionis’ Spinraza Approved by FDA for SMA

FDA has approved Ionis’ Spinraza (nusinersen), the first drug approved to treat children and adults with spinal muscular atrophy (SMA). An agency statement says FDA worked closely with Ionis during development to help design and implement the analysis upon which the approval was based.

Spinraza’s efficacy was evaluated in a trial of 121 patients with infantile-onset SMA who were diagnosed before six months of age and who were less than seven months old at the time of their first dose. The trial assessed the percentage of patients with improvement in motor milestones, such as head control, sitting, ability to kick in a supine position, rolling, crawling, standing, and walking, FDA says.

The statement says the agency asked the sponsor to conduct an interim analysis as a way to evaluate the study results as early as possible. Some 82 of the 121 patients were eligible for the analysis. Of them, it says, 40% of those treated with Spinraza achieved improvement in motor milestones, whereas none of the control patients did.

The most common side effects in the clinical trials were upper respiratory infection, lower respiratory infection, and constipation. Warnings and Precautions include low blood platelet count and renal toxicity.

FDA granted the application fast track and orphan drug designations and priority review.

Ionis is receiving a rare pediatric priority review voucher under a program FDA says is intended to encourage the development of new drugs and biologics for the prevention and treatment of rare pediatric diseases.

The notice says Spinraza will be marketed by Biogen.

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