Golodirsen - Sarepta Therapeutics

Drug Profile

Golodirsen - Sarepta Therapeutics

Alternative Names: Exon 53 PMO; SRP 4053

Latest Information Update: 24 Mar 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

  • 22 May 2018 Sarepta Therapeutics plans a phase III extension trial for Duchenne muscular dystrophy in June 2018 (NCT03532542)
  • 12 Mar 2018 Sarepta Therapeutics announces intention to submit NDA to US FDA for Duchenne muscular dystrophy by end of 2018
  • 06 Sep 2017 Interim efficacy data from a phase I/II trial in Duchenne muscular dystrophy released by Sarepta Therapeutics
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