Nusinersen - Ionis Pharmaceuticals/Biogen

Drug Profile

Nusinersen - Ionis Pharmaceuticals/Biogen

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

Latest Information Update: 21 Jun 2018

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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

  • 23 Apr 2018 Registered for Spinal muscular atrophy in Chile (Intrathecal) before April 2018
  • 23 Apr 2018 Efficacy data from the phase III SHINE, phase I/II CS2 and phase II CS12 trials in Spinal muscular atrophy released by Biogen
  • 04 Apr 2018 Biogen has patent protection for nusinersen in USA and Europe
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