Nusinersen
Nusinersen (formerly, IONIS-SMNRx, ISIS-SMNRx) is an investigational drug for spinal muscular atrophy developed by Ionis Pharmaceuticals and Biogen with financial support from SMA Foundation. It is a proprietary antisense oligonucleotide that modulates alternate splicing of the SMN2 gene, functionally converting it into SMN1 gene.
Nusinersen has orphan drug designation in the United States and the European Union.[1]
As of August 2016[update], a phase III trial in type 1 SMA patients was ended early with Biogen deciding to file for regulatory approval for nusinersen in this SMA type.[2] Another phase III trial in types 2 and 3 SMA patients is ongoing.
The drug is administered directly to the central nervous system using intrathecal injection once every 3–4 months.
In September 2016, Biogen submitted a New Drug Application to the FDA[3] and in the following month, a marketing authorisation application to European Medicines Agency under the centralised procedure.[4]
References
- ^ "nusinersen". NHS New Drugs Online Database. Retrieved 2016-05-14.
- ^ "Biogen and Ionis Pharmaceuticals Provide Important Update on First Ever SMA Regulatory Filings". CureSMA. 1 August 2016.
- ^ "Biogen Completes Rolling Submission of New Drug Application to FDA for Nusinersen as a Treatment for Spinal Muscular Atrophy". Yahoo! Finance News. 2016-09-26. Retrieved 2016-10-09.
- ^ "Biogen & Ionis submit Nusinersen application to EMA for marketing authorisation". SMA Europe.
 | This pharmacology-related article is a stub. You can help Wikipedia by expanding it. |