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Nusinersen - Biogen/Ionis Pharmaceuticals

Drug Profile

Nusinersen - Biogen/Ionis Pharmaceuticals

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

Latest Information Update: 11 May 2021

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At a glance

  • Originator Genzyme Corporation; Isis Pharmaceuticals
  • Developer Biogen; Ionis Pharmaceuticals
  • Class Antisense oligonucleotides
  • Mechanism of Action RNA interference; 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

  • 04 May 2021 The UK National Institute for Health and Care Excellence (NICE) extends clinical eligibility of nusinersen to include non-ambulant Type III spinal muscular atrophy patients under managed access agreement
  • 17 Apr 2021 Adverse events data from a part A of phase II/III trial in spinal muscular atrophy presented at the American Academy of Neurology (AAN-2021)
  • 12 Apr 2021 Biogen completes an expanded access programme for infantile-Spinal muscular atrophy (In adolescents, In children, In adults) in Colombia, New Zealand and Turkey, USA, Australia, Canada, Germany, Italy, Sweden and in the UK prior to April 2021 (NCT02865109)
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