What is "nusinersen"

Nusinersen (formerly, IONIS-SMNR, ISIS-SMNR) 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.

, 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. 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.