FDAnews Drug Daily Bulletin
Pharmaceuticals / Submissions and Approvals

FDA Approves Biogen’s Spinraza as First Treatment for Spinal Muscular Atrophy

Jan. 6, 2017
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FDA approved Biogen’s Spinraza as the first treatment for spinal muscular atrophy in pediatric and adult patients.

The approval was based on a clinical trial of 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. Additional open-label uncontrolled clinical studies were conducted in symptomatic patients who ranged in age from 30 days to 15 years at the time of the first dose.

Spinraza was previously granted fast track and priority review by the FDA, and is currently undergoing accelerated review at the European Medicines Agency. It was developed by Ionis Pharmaceuticals.

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