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Drisapersen

Drug Profile

Drisapersen

Alternative Names: 2'-O-methyl-phosphorothioate oligonucleotide; 2402968; GSK-2402968; GSK2402968A; h51AON23; Kyndrisa; PRO-051

Latest Information Update: 04 Nov 2018

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At a glance

  • Originator Prosensa
  • Developer BioMarin Nederland
  • Class Antisense oligonucleotides
  • Mechanism of Action Dystrophin stimulants; RNA interference
  • Orphan Drug Status

    Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

    Yes - Duchenne muscular dystrophy
  • New Molecular Entity Yes

Highest Development Phases

  • No development reported Duchenne muscular dystrophy

Most Recent Events

  • 24 Jun 2018 Biomarkers information updated
  • 21 Jul 2016 BioMarin Pharmaceuticals completes the phase III Drisapersen confirmatory trial for Duchenne muscular dystrophy (In children, In adolescents) in Belgium (SC) (EudraCT2014-005296-81)
  • 31 May 2016 Discontinued - Phase-III for Duchenne muscular dystrophy in Netherlands, Spain (SC)
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