Nusinersen: Difference between revisions
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'''Nusinersen''' (formerly, IONIS-SMN<sub>Rx</sub>, ISIS-SMN<sub>Rx</sub>), marketed as Spinraza,<ref>{{cite web|url=http://smanewstoday.com/2016/11/01/regulatory-applications-sma-therapy-nusinersen-accepted-us-fda-eu-ema |title=Regulatory Applications for SMA Therapy Nusinersen Accepted in US, EU |publisher=BioNews Services, LLC|date= |accessdate=2016-11-15}}</ref> is the first drug approved by the U.S. [[Food and Drug Administration]] for use in treating [[spinal muscular atrophy]] (SMA), a rare pediatric disease. |
'''Nusinersen''' (formerly, IONIS-SMN<sub>Rx</sub>, ISIS-SMN<sub>Rx</sub>), marketed as Spinraza,<ref>{{cite web|url=http://smanewstoday.com/2016/11/01/regulatory-applications-sma-therapy-nusinersen-accepted-us-fda-eu-ema |title=Regulatory Applications for SMA Therapy Nusinersen Accepted in US, EU |publisher=BioNews Services, LLC|date= |accessdate=2016-11-15}}</ref> is the first drug approved by the U.S. [[Food and Drug Administration]] for use in treating [[spinal muscular atrophy]] (SMA), a rare pediatric disease. It was developed by [[Ionis Pharmaceuticals]] and licensed to [[Biogen]] for a $75 million license fee,<ref name="genengnews_2016">{{citation |url=http://www.genengnews.com/gen-news-highlights/biogen-shells-out-75m-to-develop-ionis-nusinersen-after-positive-phase-iii-results/81253027 |date=August 1, 2016 |title=Biogen Shells Out $75M to Develop Ionis' Nusinersen after Positive Phase III Results |accessdate=December 31, 2016}}</ref> with initial financial support from SMA Foundation and Cure SMA. |
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== Medical use== |
== Medical use== |
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==Pharmacology== |
==Pharmacology== |
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spinal muscular atrophy is caused by mutations that cause the ''[[SMN1]]'' gene to not function. Nusinersen is a proprietary [[antisense oligonucleotide]] that modulates [[alternate splicing]] of the ''[[SMN2]]'' gene, functionally converting it into ''SMN1'' gene.<ref>{{cite journal|last1=Zanetta|first1=C|last2=Nizzardo|first2=M|last3=Simone|first3=C|last4=Monguzzi|first4=E|last5=Bresolin|first5=N|last6=Comi|first6=GP|last7=Corti|first7=S|title=Molecular therapeutic strategies for spinal muscular atrophies: current and future clinical trials.|journal=Clinical therapeutics|date=1 January 2014|volume=36|issue=1|pages=128-40|doi=10.1016/j.clinthera.2013.11.006|pmid=24360800}}</ref> |
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==History== |
==History== |
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Nusinersen (formerly, IONIS-SMNRx, ISIS-SMNRx), marketed as Spinraza,[1] is the first drug approved by the U.S. Food and Drug Administration for use in treating spinal muscular atrophy (SMA), a rare pediatric disease. It was developed by Ionis Pharmaceuticals and licensed to Biogen for a $75 million license fee,[2] with initial financial support from SMA Foundation and Cure SMA.
Medical use
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.
Side effects
Pharmacology
spinal muscular atrophy is caused by mutations that cause the SMN1 gene to not function. Nusinersen is a proprietary antisense oligonucleotide that modulates alternate splicing of the SMN2 gene, functionally converting it into SMN1 gene.[3]
History
Society and culture
Nusinersen has orphan drug designation in the United States and the European Union.[5]
Biogen paid a $75 million license fee to Ionis Pharmaceuticals in August 2016, "after exercising its option to develop and commercialize nusinersen globally"[2] The drug was accepted as a FDA's priority review drug 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."[6] 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."[6] 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."[7]
References
- ^ "Regulatory Applications for SMA Therapy Nusinersen Accepted in US, EU". BioNews Services, LLC. Retrieved 2016-11-15.
- ^ a b Biogen Shells Out $75M to Develop Ionis' Nusinersen after Positive Phase III Results, August 1, 2016, retrieved December 31, 2016
- ^ Zanetta, C; Nizzardo, M; Simone, C; Monguzzi, E; Bresolin, N; Comi, GP; Corti, S (1 January 2014). "Molecular therapeutic strategies for spinal muscular atrophies: current and future clinical trials". Clinical therapeutics. 36 (1): 128–40. doi:10.1016/j.clinthera.2013.11.006. PMID 24360800.
- ^ Grant, Charley (2016-12-27). "Surprise Drug Approval Is Holiday Gift for Biogen". Wall Street Journal. ISSN 0099-9660. Retrieved 2016-12-27.
- ^ "nusinersen". NHS New Drugs Online Database. Retrieved 2016-05-14.
- ^ a b 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) - ^ Cory Renauer (October 5, 2016), Better Buy: Anavex Life Sciences Corp. vs. Ionis Pharmaceuticals, Inc., retrieved December 31, 2016
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