Biogen and IONIS Pharmaceuticals have announced that the antisense oligonucleotide therapy nusinersen has met the primary endpoint following interim analysis of the Phase III double-blind, randomized clinical trial to treat infant onset spinal muscular atrophy (SMA): the drug significantly improved achievement of motor milestones in treated as compared to untreated infants. Nusinersen, previously called IONIS-SMN Rx, increases levels of the survival of motor neuron (SMN) protein by modulating splicing of SMN2, a gene nearly identical to the SMN1 gene mutated in SMA. Biogen has exercised its option to license and commercialize the technology from IONIS (see Dec 2014 news), and is planning to apply for regulatory approval in the US this year. If approved, this would be the first FDA-approved drug to treat SMA.
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