Golodirsen - Sarepta Therapeutics

Drug Profile

Golodirsen - Sarepta Therapeutics

Alternative Names: Exon 53 PMO; SRP 4053

Latest Information Update: 13 Sep 2017

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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

  • 06 Sep 2017 Interim efficacy data from a phase I/II trial in Duchenne muscular dystrophy released by Sarepta Therapeutics
  • 18 Jul 2017 Sarepta Therapeutics and BioMarin Pharmaceutical execute a global settlement and a license agreement resolving exon skipping patent litigation
  • 18 Jul 2017 Sarepta Therapeutics and BioMarin Pharmaceutical execute a settlement agreement to resolve global patent proceedings regarding Sarepta’s sale of eteplirsen and future Duchenne muscular dystrophy exon-skipping products
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