The Food and Drug Administration (FDA) has approved Spinraza (nusinersen; Biogen) intrathecal injection under Priority Review for the treatment of spinal muscular atrophy (SMA) in children and adults. This approval marks the first treatment approved in the United States for SMA. 

The approval was based on positive data from multiple clinical studies involving >170 patients. ENDEAR, a Phase 3 randomized, double-blind, sham-controlled study, enrolled patients with infantile-onset SMA. At the planned interim analysis, a greater percentage of infants who received Spinraza achieved a motor milestone vs. patients who did not receive treatment (40% vs. 0%; P<0.0001) according to the Hammersmith Infant Neurological Examination (HINE). Some milestones included the ability to sit unassisted, stand or walk when patients would otherwise be unexpected to do so. Also, a smaller percentage of patients in the Spinraza group died vs. the untreated patients (23% vs. 43%). Patients who received Spinraza also demonstrated positive data for other efficacy endpoints that were evaluated. 

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Spinraza, an antisense oligonucleotide (ASO), is intended to treat SMA caused by mutations in the chromosome 5q that leads to SMN protein deficiency. It works by altering the splicing of SMN2 pre-mRNA in order to increase production of full-length SMN protein. Spinraza can potentially increase the amount of full-length SMN protein in patients with SMA.

Spinraza will be available as a 12mg/5mL strength preservative-free solution in a single-dose glass vial. It is anticipated to be available in approximately one week.

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