Golodirsen - Sarepta Therapeutics

Drug Profile

Golodirsen - Sarepta Therapeutics

Alternative Names: Exon 53 PMO; SRP 4053

Latest Information Update: 11 Oct 2018

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At a glance

  • Originator Sarepta Therapeutics
  • Class Antisense oligonucleotides; Morpholines
  • Mechanism of Action Dystrophin expression modulators; Genetic transcription inhibitors
  • Orphan Drug Status

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

  • New Molecular Entity Yes

Highest Development Phases

  • Phase III Duchenne muscular dystrophy

Most Recent Events

  • 02 Aug 2018 Phase-III clinical trials in Duchenne muscular dystrophy (In adolescents, In adults, In children) in Spain, United Kingdom, Italy, USA (IV) (NCT03532542) (EudraCT2017-004625-32)
  • 24 Jun 2018 Biomarkers information updated
  • 22 May 2018 Sarepta Therapeutics plans a phase III extension trial for Duchenne muscular dystrophy in June 2018 (NCT03532542)
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