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Nusinersen - Biogen/Ionis Pharmaceuticals

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Drug Profile

Nusinersen - Biogen/Ionis Pharmaceuticals

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

Latest Information Update: 26 Feb 2026

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At a glance

  • Originator Cold Spring Harbor Laboratory; 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

  • 16 Feb 2026 Nusinersen is covered by patent protection in USA and Europe
  • 12 Jan 2026 European Commission approves high-dose regimen nusinersen for treatment of Spinal muscular atrophy in European Union
  • 17 Nov 2025 Nusinersen receives positive CHMP opinion from the European Medicines Agency for 5q Spinal muscular atrophy

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