Nusinersen

K K Jain MD (Dr. Jain is a consultant in neurology and has no relevant financial relationships to disclose.)
Originally released March 8, 2017; expires March 8, 2020

This article includes discussion of BIIB058, Ionis-SMNRx, ISIS 396443, and ISIS-SMNRx. The foregoing terms may include synonyms, similar disorders, variations in usage, and abbreviations.

Historical note and terminology

Nusinersen is a splice-modulating antisense oligonucleotide indicated for the treatment of spinal muscular atrophy. Intrathecal nusinersen was approved by the U.S. Food and Drug Administration (FDA) in December 2016. Another splice-modulating oligonucleotide, eteplirsen, was also approved earlier in 2016 for the treatment of Duchenne muscular dystrophy. The FDA granted fast track designation and priority review to the application for nusinersen. The drug also received orphan drug designation, which provides incentives to assist and encourage the development of drugs for rare diseases. Pharmacological data and results of clinical trials submitted for the approval of nusinersen can be viewed on the FDA website. Regulatory assessments for nusinersen as a treatment for spinal muscular atrophy are underway in the European Union and in several other countries (Hoy 2017).

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