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'''Nusinersen''' (formerly, IONIS-SMN<sub>Rx</sub>, ISIS-SMN<sub>Rx</sub>), intended to be 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 an investigational drug for ] developed by ] and ] with financial support from ] and ]. It is a proprietary ] that modulates ] of the '']'' gene, functionally converting it into '']'' gene. '''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 ] for use in treating ]. Nusinersen is a proprietary ] that modulates ] of the '']'' gene, functionally converting it into '']'' gene. It has been developed by ] and licensed to ], with initial financial support from ] and ].


The drug is administered directly to the ] using ] injection once every 3–4 months. The drug is authorised for use in all types of spinal muscular atrophy regardless of age and disease stage. It is administered directly to the ] using ] injection once every 4 months.


Nusinersen has ] designation in the ] and the ].<ref>{{cite web|url=http://www.ukmi.nhs.uk/applications/ndo/record_view_open.asp?newDrugID=6236 |title=nusinersen |publisher=NHS New Drugs Online Database |date= |accessdate=2016-05-14}}</ref> Nusinersen has ] designation in the ] and the ].<ref>{{cite web|url=http://www.ukmi.nhs.uk/applications/ndo/record_view_open.asp?newDrugID=6236 |title=nusinersen |publisher=NHS New Drugs Online Database |date= |accessdate=2016-05-14}}</ref>

In August 2016, a phase III trial in type 1 SMA patients was ended early due to positive efficacy data, with Biogen deciding to file for regulatory approval for the drug.<ref>{{cite web | url=http://www.curesma.org/news/biogen-and-ionis-august-2016-statement.html | title=Biogen and Ionis Pharmaceuticals Provide Important Update on First Ever SMA Regulatory Filings | publisher=CureSMA | date=1 August 2016 }}</ref> Consequently, the company submitted a ] to the ] in September 2016<ref>{{Cite news|url=http://finance.yahoo.com/news/biogen-completes-rolling-submission-drug-113000831.html|title=Biogen Completes Rolling Submission of New Drug Application to FDA for Nusinersen as a Treatment for Spinal Muscular Atrophy|last=|first=|date=2016-09-26|work=Yahoo! Finance News|access-date=2016-10-09|via=}}</ref> and a marketing authorisation application to the ], under the ],<ref>{{cite web | url=http://www.sma-europe.eu/news/nusinersen-application-to-ema | title=Biogen & Ionis submit Nusinersen application to EMA for marketing authorisation | publisher=SMA Europe }}</ref> in the following month. The company also announced an ] of nusinersen in type 1 SMA in selected countries.

In November 2016, a phase III clinical trial in type 2 SMA patients was halted after an interim analysis indicated the drug's efficacy also in this SMA type.<ref>{{cite web| url=http://www.alzforum.org/news/research-news/positive-trials-spinal-muscular-atrophy-bode-well-antisense-approach | title=Positive Trials of Spinal Muscular Atrophy Bode Well for Antisense Approach| publisher=] }}</ref>


== References == == References ==

Revision as of 16:05, 24 December 2016

Nusinersen (formerly, IONIS-SMNRx, ISIS-SMNRx), marketed as Spinraza, is the first drug approved by 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 has been developed by Ionis Pharmaceuticals and licensed to Biogen, with initial financial support from SMA Foundation and Cure SMA.

The drug is authorised 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. "nusinersen". NHS New Drugs Online Database. Retrieved 2016-05-14.
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