Nusinersen

Drug Profile

Nusinersen

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

Latest Information Update: 11 Jan 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
  • On Fast track

    Fast track status is assigned by the US FDA so therapies with the potential to address unmet needs can move faster through development.

    No
  • New Molecular Entity Yes

Highest Development Phases

  • Registered Spinal muscular atrophy

Most Recent Events

  • 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
  • 23 Dec 2016 Registered for Spinal muscular atrophy (In adolescents, In children, In infants, In adults, In neonates) in USA (Intrathecal) - First global approval
  • 23 Dec 2016 The US FDA issues rare paediatric disease priority review voucher to Ionis Pharmaceuticals
Restricted Access

Oops, it looks like you don’t have a valid subscription to this content. To gain full access to the content and functionality of the AdisInsight database try one of the following.

  • with a username/password associated to an account with a valid subscription
  • Contact your organization’s admin about adding this content to your AdisInsight subscription
  • Request a trial

If you are a subscriber to this content then contact us at AsktheExpert.AdisInsight@springer.com so we can help.

Back to top