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A Phase I/II, Open Label, Escalating Dose, Pilot Study to Assess the Effect, Safety, Tolerability and Pharmacokinetics of Multiple Subcutaneous Doses of Drisapersen in Patients With Duchenne Muscular Dystrophy and to Assess the Potential for Intravenous Dosing as an Alternative Route of Administration

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Trial Profile

A Phase I/II, Open Label, Escalating Dose, Pilot Study to Assess the Effect, Safety, Tolerability and Pharmacokinetics of Multiple Subcutaneous Doses of Drisapersen in Patients With Duchenne Muscular Dystrophy and to Assess the Potential for Intravenous Dosing as an Alternative Route of Administration

Status: Discontinued
Phase of Trial: Phase I/II

Latest Information Update: 10 Dec 2023

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At a glance

  • Drugs Drisapersen (Primary)
  • Indications Duchenne muscular dystrophy
  • Focus Adverse reactions; Pharmacokinetics; Registrational
  • Sponsors BioMarin Nederland; BioMarin Pharmaceutical; GlaxoSmithKline

Most Recent Events

  • 01 Feb 2019 Results published in the European Journal of Pediatrics
  • 31 May 2016 According to a BioMarin media release, the company has withdrawn its MAA for drisapersen (Kyndrisa) from the EMA following discussions at the May 2016 CHMP meeting.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 active, no longer recruiting to discontinued, according to a BioMarin media release.

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