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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

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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: 09 Feb 2024

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At a glance

  • Drugs Valoctocogene-roxaparvovec (Primary)
  • Indications Haemophilia A
  • Focus Adverse reactions; First in man; Pharmacodynamics; Registrational
  • Acronyms GENEr8-INH
  • Sponsors BioMarin Pharmaceutical

    • Most Recent Events

    • 06 Feb 2024 According to a BioMarin Pharmaceutical Media Release, 7-year follow-up data from this study will be presented at the 2024 European Association for Haemophilia and Allied Disorders (EAHAD) Congress, Feb. 6-9, 2024.
    • 06 Feb 2024 Preliminary results presented in a BioMarin Pharmaceutical Media Release.
    • 23 Nov 2022 According to a BioMarin Pharmaceutical media release, the Company was recently notified by the FDA that after further consideration, at this time, the Agency no longer plans to hold an advisory committee meeting to discuss the BLA for ROCTAVIAN that is currently under review. Previously, the FDA communicated to the Company that it did intend to hold an advisory committee meeting but did not specify a date.
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