Nusinersen- Ionis Pharmaceuticals/Biogen

Drug Profile

Nusinersen- Ionis Pharmaceuticals/Biogen

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

Latest Information Update: 24 Mar 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 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

  • 12 Mar 2018 Interim efficacy and adverse events data from the phase II NURTURE trial in Spinal muscular atrophy released by Biogen
  • 22 Jan 2018 Biogen intends to submit Spinraza® (nusinersen) for the recommendation on whether routine funding can be made available to patients for Spinal muscular atrophy in United Kingdom via the NHS to NICE, in March 2018
  • 22 Jan 2018 The UK National Institute for Health and Care Excellence (NICE) invites Biogen to submit Spinraza® (nusinersen) for Spinal muscular atrophy, for assessment via the Single Technology Appraisal (STA) route
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