In December 2016, the US Food and Drug Administration (FDA) approved nusinersen sodium for the treatment of spinal muscular atrophy (SMA), a rare genetic disease with an incidence of 1 in 6000 to 10 000 live births. The approval was supported by an interim analysis of a randomized, blinded, sham-controlled trial, whose final results were published in 2017.1,2 Policy debates have centered on the cost of nusinersen, which has a list price of $750 000 for the first year of treatment (4 doses administered intrathecally during the first 10 weeks, followed by maintenance doses every 4 months).3 There has been less discussion of the limited evidence supporting the drug’s efficacy and potential harms.
Gerrity MS, Prasad V, Obley AJ. Concerns About the Approval of Nusinersen Sodium by the US Food and Drug Administration. JAMA Intern Med. 2018;178(6):743–744. doi:10.1001/jamainternmed.2018.0869
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