An open-label extension study of the long-term safety, tolerability and efficacy of drisapersen in subjects with Duchenne Muscular Dystrophy.

Trial Profile

An open-label extension study of the long-term safety, tolerability and efficacy of drisapersen in subjects with Duchenne Muscular Dystrophy.

Discontinued
Phase of Trial: Phase III

Latest Information Update: 08 May 2017

At a glance

  • Drugs Drisapersen (Primary) ; Drisapersen (Primary)
  • Indications Duchenne muscular dystrophy
  • Focus Adverse reactions; Registrational; Therapeutic Use
  • Sponsors BioMarin Pharmaceutical
  • Most Recent Events

    • 05 May 2017 This trial has been completed in Germany (End date:2016-09-09) as per European Clinical Trials Database record.
    • 31 May 2016 According to a BioMarin media release, the company has withdrawn its MAA for drisapersen (Kyndrisa) from the EMA following discussions at the May 2016 CHMP meeting. Those discussions clearly indicated that the CHMP intended to issue a negative opinion. BioMarin intends to discontinue clinical and regulatory development of Kyndrisa as well as the three other first-generation follow-on products, BMN 044, BMN 045 and BMN 053.
    • 31 May 2016 Status changed from recruiting to discontinued, according to a BioMarin media release.
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