An Open-Label, Multi-Center Study to Evaluate the Safety and Tolerability of Eteplirsen in Patients With Advanced Stage Duchenne Muscular Dystrophy

Trial Profile

An Open-Label, Multi-Center Study to Evaluate the Safety and Tolerability of Eteplirsen in Patients With Advanced Stage Duchenne Muscular Dystrophy

Active, no longer recruiting
Phase of Trial: Phase II

Latest Information Update: 30 Mar 2017

At a glance

  • Drugs Eteplirsen (Primary)
  • Indications Duchenne muscular dystrophy
  • Focus Adverse reactions; Registrational
  • Sponsors Sarepta Therapeutics
  • Most Recent Events

    • 10 Jan 2017 Time frame of primary endpoint has changed from 96 weeks up to 144 weeks.
    • 19 Sep 2016 According to CureDuchenne Ventures media release, eteplirsen received accelerated approval from the Food and Drug Administration to treat Duchenne muscular dystrophy.
    • 25 May 2016 According to a Sarepta Therapeutics media release, the US FDA has informed the company that they will not be able to complete the review of eteplirsen NDA by the Prescription Drug User Fee Act (PDUFA) goal date of May 26, 2016.
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