Nusinersen

Drug Profile

Nusinersen

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

Latest Information Update: 20 May 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

  • 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)
  • 21 Apr 2017 Committee for Medicinal Products for Human Use (CHMP) of the EMA recommends approval of nusinersen for Spinal muscular atrophy (In adolescents, In adults, In children, In infants, In neonates) in European Union
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