Misplaced Pages

This is an old revision of this page, as edited by Natureium (talk | contribs) at 19:48, 27 December 2016 (copyediting). The present address (URL) is a permanent link to this revision, which may differ significantly from the current revision.

Nusinersen (formerly, IONIS-SMN_Rx, ISIS-SMN_Rx), marketed as Spinraza, is the first drug approved by the U.S. Food and Drug Administration for use in treating spinal muscular atrophy. Nusinersen is a proprietary antisense oligonucleotide that modulates alternate splicing of the SMN2 gene, functionally converting it into SMN1 gene. It was developed by Ionis Pharmaceuticals and licensed to Biogen, with initial financial support from SMA Foundation and Cure SMA.

Status

The drug is approved for use in all types of spinal muscular atrophy regardless of age and disease stage. It is administered directly to the central nervous system using intrathecal injection once every 4 months.

Nusinersen has orphan drug designation in the United States and the European Union.

References

1. "Regulatory Applications for SMA Therapy Nusinersen Accepted in US, EU". BioNews Services, LLC. Retrieved 2016-11-15.
2. Grant, Charley (2016-12-27). "Surprise Drug Approval Is Holiday Gift for Biogen". Wall Street Journal. ISSN 0099-9660. Retrieved 2016-12-27.
3. "nusinersen". NHS New Drugs Online Database. Retrieved 2016-05-14.

This pharmacology-related article is a stub. You can help Misplaced Pages by expanding it.

• v
• t
• e

• Pharmacology stubs
• Orphan drugs
• Drugs acting on the nervous system
• Experimental drugs