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A Phase 1/2, Dose-Escalation Safety, Tolerability and Efficacy Study of BMN 270, an Adenovirus-Associated Virus Vector-Mediated Gene Transfer of Human Factor VIII in Patients with Severe Haemophilia A

Trial Profile

A Phase 1/2, Dose-Escalation Safety, Tolerability and Efficacy Study of BMN 270, an Adenovirus-Associated Virus Vector-Mediated Gene Transfer of Human Factor VIII in Patients with Severe Haemophilia A

Status: Active, no longer recruiting
Phase of Trial: Phase I/II

Latest Information Update: 08 Nov 2019

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At a glance

  • Drugs Valoctocogene-roxaparvovec (Primary)
  • Indications Haemophilia A
  • Focus Adverse reactions; First in man; Pharmacodynamics
  • Sponsors BioMarin Pharmaceutical
  • Most Recent Events

    • 23 Oct 2019 According to a BioMarin Pharmaceutical media release, on July 8, the Company announced that based on recent meetings with health authorities in the U.S. and Europe, it plans to submit marketing applications to both the FDA and the EMA in the fourth quarter of 2019 for valoctocogene roxaparvovec with the 6e13 vg/kg dose. The submissions will be based on the recently completed Phase 3 interim analysis and the updated three-year Phase 1/2 data of patients treated with valoctocogene roxaparvovec.
    • 23 Oct 2019 According to a BioMarin Pharmaceutical media release, company intends to provide a 4 year update with the 6e13 vg/kg dose subjects and a 3 year update with the 4e13 vg/kg dose subjects from the ongoing Phase 2 study in mid-2020.
    • 08 Jul 2019 According to a BioMarin Pharmaceutical media release, data were presented at the 27th International Society on Thrombosis and Haemostasis (ISTH) 2019 Congress.
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