Biogen and IONIS Pharmaceuticals have submitted a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for the approval of nusinersen, an investigational treatment for spinal muscular atrophy (SMA). Nusinersen is an antisense oligonucleotide that is designed to modify the splicing of survival of motor neuron 2 (SMN2) gene, a gene closely related to the mutated SMN1 gene that causes SMA. Nusinersen treatment was found to significantly improve achievement of motor milestones following interim analysis of the ongoing Phase III clinical trial (see Aug 2016 news). In addition to the NDA submission to the FDA, Biogen plans to submit a Marketing Authorization Application to the European Medicines Agency in the coming weeks.
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