Drug Profile


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

Latest Information Update: 25 Mar 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

  • 26 Jan 2017 Launched for Spinal muscular atrophy (In adolescents, In children, In infants, In neonates, In adults) in USA (Intrathecal)
  • 13 Jan 2017 End-of-study efficacy and adverse events data from the phase III ENDEAR trial in Spinal muscular atrophy released by Biogen
  • 23 Dec 2016 Preregistration for Spinal muscular atrophy (In adolescents, In children, In infants, In adults, In neonates) in Japan, Canada, Australia (Intrathecal) before December 2016
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