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A Phase 3 Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Evaluate the Efficacy and Safety of BMN 111 in Children with Achondroplasia

Trial Profile

A Phase 3 Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Evaluate the Efficacy and Safety of BMN 111 in Children with Achondroplasia

Status: Completed
Phase of Trial: Phase III

Latest Information Update: 11 Sep 2024

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At a glance

  • Drugs Vosoritide (Primary)
  • Indications Achondroplasia
  • Focus Registrational; Therapeutic Use
  • Sponsors BioMarin Pharmaceutical
  • Most Recent Events

    • 04 Sep 2024 According to a BioMarin Pharmaceutical media release, the Company expects data readout in 2026, and potential approval in 2027.
    • 21 Jun 2022 According to a BioMarin Pharmaceutical media release, the Ministry of Health, Labor and Welfare (MHLW) in Japan granted approval of the registration of VOXZOGO (vosoritide) for injection, indicated for the treatment of achondroplasia in children of all ages, whose growth plates are not closed.
    • 23 Feb 2022 Updated results published in the BioMarin Pharmaceutical financial media release.

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