Nusinersen- Ionis Pharmaceuticals/Biogen

Drug Profile

Nusinersen- Ionis Pharmaceuticals/Biogen

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

Latest Information Update: 15 Nov 2017

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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

  • 07 Nov 2017 Nusinersen is available on a Named Patient Access Programme for the treatment of Spinal muscular atrophy in Middle East and Latin America
  • 01 Nov 2017 Registered for Spinal muscular atrophy (In adolescents, In children, In infants, In neonates, In adults) in Switzerland (Intrathecal), before November 2017
  • 01 Nov 2017 Final efficacy data from the phase III ENDEAR trial in Spinal muscular atrophy released by Biogen
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