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Eteplirsen - Sarepta Therapeutics

Drug Profile

Eteplirsen - Sarepta Therapeutics

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

Latest Information Update: 08 Jul 2019

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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

  • 20 Jun 2019 Sarepta Therapeutics plans a phase III trial for Duchenne muscular dystrophy (Treatment-experienced) (IV) (NCT03992430)
  • 19 Jun 2019 Sarepta Therapeutics plans a phase II trial for Duchenne muscular dystrophy (IV) (NCT03985878)
  • 14 Jun 2019 Sarepta Therapeutics completes the phase III PROMOVI trial in Duchenne muscular dystrophy in USA (IV) (NCT02255552)
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