Final Analysis of the Phase 1/2 Trial of Valoctocogene Roxaparvovec for Severe Haemophilia A
INTRODUCTION: Valoctocogene roxaparvovec is an adeno-associated virus vector serotype 5 (AAV5)-mediated gene therapy for severe haemophilia A (HA). AIM: Report the final safety and efficacy results of the phase 1/2 trial of valoctocogene roxaparvovec. METHODS: vg/kg cohort results were reported previously. RESULTS: participants remain off prophylaxis. Haemo-QOL-A Total Score was mostly stable from baseline. The last treatment-related AE (TRAE) of alanine aminotransferase elevation occurred in year 1. No serious TRAEs occurred after year 1. No participants had thromboembolic events or developed FVIII inhibitors. CONCLUSION: vg/kg. No concerning long-term safety signals were identified.
Authors
- Savita Rangarajan (ORCID: https://orcid.org/0000-0001-7367-133X)
- Emily Symington (ORCID: https://orcid.org/0000-0003-2529-8168)
- Mingjin Li
- Glenn F. Pierce (ORCID: https://orcid.org/0000-0002-3310-328X)
- Carolyn M. Millar
- Dane Osmond
- Konstantia-Maria Chavele
- Bella Madan (ORCID: https://orcid.org/0000-0003-4858-4888)
- P Raheja
- Will Lester
Institutions
- University Hospitals Birmingham NHS Foundation Trust (GB)
- Imperial College Healthcare NHS Trust (GB)
- Guy's and St Thomas' NHS Foundation Trust (GB)
- Barts Health NHS Trust (GB)
- Cambridge University Hospitals NHS Foundation Trust (GB)
- Museum of Contemporary Art San Diego (US)
- University of Southampton (GB)
- BioMarin (United States) (US)
Publication Details
- Journal
- Haemophilia
- Published
- 2026-04-12
- DOI
- https://doi.org/10.1111/hae.70284
- Primary Topic
- Hemophilia Treatment and Research
- Type
- article
- Field-Weighted Citation Impact
- 0.00
Funders
- BioMarin Pharmaceutical