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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.

In December 2016 the F.D.A. approved the drug Spinraza, also known as nusinersen which treats spinal muscular atrophy, a rare pediatric disease. Biogen, which is licensing Spinraza from Ionis Pharmaceuticals, benefited from the FDA's priority review because of a successful 2015 clinical trial involving 82 infants in which "40 percent of babies on the drug reached milestones such as sitting, crawling and walking." According to the New York Times, Spinraza, "will be among the most expensive drugs in the world", with an estimated cost $750,000 in the first year of treatment and "about $375,000 annually after that." Peak annual sales of nusinersen "could reach around $1.5 billion, triggering up to $150 million in additional milestone payments and a royalty percentage up to the mid-teens."

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.
4. ^ Katie Thomas (December 30, 2016), Costly Drug for Fatal Muscular Disease Wins F.D.A. Approval, New York Times, retrieved December 31, 2016 {{citation}}: Italic or bold markup not allowed in: |publisher= (help)
5. Cory Renauer (October, 2016), Better Buy: Anavex Life Sciences Corp. vs. Ionis Pharmaceuticals, Inc., retrieved December 31, 2016 {{citation}}: Check date values in: |date= (help)

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