Nusinersen

Nusinersen (formerly, IONIS-SMN_Rx, ISIS-SMN_Rx), also known by trade name SPINRAZA,^[1] is an investigational drug for spinal muscular atrophy developed by Ionis Pharmaceuticals and Biogen with financial support from SMA Foundation and Cure SMA. It is a proprietary antisense oligonucleotide that modulates alternate splicing of the SMN2 gene, functionally converting it into SMN1 gene.

The drug is administered directly to the central nervous system using intrathecal injection once every 3–4 months.

Nusinersen has orphan drug designation in the United States and the European Union.^[2]

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.^[3] Consequently, the company submitted a New Drug Application to the FDA in September 2016^[4] and a marketing authorisation application to the European Medicines Agency, under the centralised procedure,^[5] in the following month. The company also announced an expanded access programme 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.^[6]

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.
3. "Biogen and Ionis Pharmaceuticals Provide Important Update on First Ever SMA Regulatory Filings". CureSMA. 1 August 2016.
4. "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.
5. "Biogen & Ionis submit Nusinersen application to EMA for marketing authorisation". SMA Europe.
6. "Positive Trials of Spinal Muscular Atrophy Bode Well for Antisense Approach". alzforum.org.