US FDA approves first treatment for spinal muscular atrophy

27 December 2016

The US Food and Drug Administration has approved Spinraza (nusinersen) under Priority Review for the treatment of spinal muscular atrophy (SMA) in pediatric and adult patients.

Spinraza is the first and only treatment approved in the US for SMA, a leading genetic cause of death in infants and toddlers that is marked by progressive, debilitating muscle weakness. The marketing clearance was effected within three months of regulatory filing.

Nusinersen was discovered by Ionis Pharmaceuticals (Nasdaq: IONS) and licensed to biotech major Biogen (Nasdaq: BIIB). Shares of Ionis, which is eligible for a $150 million milestone payment for the approval, gained 8.3% to $53.41 in after-hours trading on Friday and having risen as much as 21%, while Biogen improved 3.6% to $287.53.

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