Drug Profile


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

Latest Information Update: 05 May 2017

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
  • New Molecular Entity Yes
  • Available For Licensing Yes

Highest Development Phases

  • Marketed Duchenne muscular dystrophy

Most Recent Events

  • 31 Mar 2017 Launched for Duchenne muscular dystrophy in USA prior to March 2017 (IV) - First global launch
  • 28 Feb 2017 Sarepta Therapeutics has patent protection for Eteplirsen in USA and EU (Sarepta therapeutics Form 10-K, March 2017)
  • 21 Feb 2017 Sarepta Therapeutics enters into an agreement to sell its Rare Paediatric Disease Priority Review Voucher
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