Drug Profile


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

Latest Information Update: 22 Dec 2016

Price : $50

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
  • On Fast track

    Fast track status is assigned by the US FDA so therapies with the potential to address unmet needs can move faster through development.

  • New Molecular Entity Yes
  • Available For Licensing Yes

Highest Development Phases

  • Registered Duchenne muscular dystrophy

Most Recent Events

  • 19 Dec 2016 Preregistration for Duchenne muscular dystrophy in European Union (IV) before December 2016
  • 20 Sep 2016 Adverse events data from a clinical trial in Duchenne muscular dystrophy released by Sarepta Therapeutics
  • 19 Sep 2016 Registered for Duchenne muscular dystrophy in USA (IV) - First global approval
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