Drug Profile


Alternative Names: AVI 4658; AVI-4658 PMO; Exondys 51

Latest Information Update: 28 Jul 2017

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At a glance

  • Originator AVI BioPharma; Ercole Biotech
  • Developer Sarepta Therapeutics
  • Class Antisense oligonucleotides; Morpholines
  • Mechanism of Action Dystrophin expression modulators; 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
  • Available For Licensing Yes

Highest Development Phases

  • Marketed Duchenne muscular dystrophy

Most Recent Events

  • 19 Jul 2017 Sarepta Therapeutics initiates a managed-access programme for Duchenne Muscular Dystrophy in Europe, North America and South America
  • 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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