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Nusinersen - Ionis Pharmaceuticals/Biogen

Drug Profile

Nusinersen - Ionis Pharmaceuticals/Biogen

Alternative Names: BIIB 058; IONIS-SMNRx; ISIS-396443; ISIS-SMNRx; Spinraza

Latest Information Update: 30 Jan 2019

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At a glance

  • Originator Genzyme Corporation; Isis Pharmaceuticals
  • Developer Biogen; Ionis Pharmaceuticals
  • Class Antisense oligonucleotides; Spinal muscular atrophy gene therapies
  • Mechanism of Action Gene transference; Survival of motor neuron 2 protein expression stimulants
  • Orphan Drug Status

    Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

    Yes - Spinal muscular atrophy
  • New Molecular Entity Yes

Highest Development Phases

  • Marketed Spinal muscular atrophy

Most Recent Events

  • 06 Oct 2018 Interim safety and efficacy data from the phase II NURTURE trial in Spinal muscular dystrophy released by Biogen
  • 06 Oct 2018 Pharmacodynamics data from clinical trials in Spinal muscular dystrophy released by Biogen
  • 24 Sep 2018 Biogen completes the phase II EMBRACE trial in Spinal muscular atrophy (In adolescents, In children, In infants, In neonates) in USA and Germany (Intrathecal) (NCT02462759)
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