Nusinersen

Drug Profile

Nusinersen

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

Latest Information Update: 05 Jun 2017

Price : $50

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

  • 01 Jun 2017 Registered for Spinal muscular atrophy (In children, In infants, In neonates) in European Union, Iceland, Norway and Liechtenstein (Intrathecal)
  • 24 Apr 2017 Final efficacy and adverse events data from a phase III CHERISH trial in Spinal muscular atrophy released by Biogen
  • 21 Apr 2017 Preregistration for Spinal muscular atrophy (In children, In adults, In infants, In adolescents, In neonates) in Switzerland (Intrathecal)
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