Phase 1/2 trial assesses valoctocogene roxaparvovec for haemostasis in severe haemophilia A, showing positive outcomes.
Over 7 years, valoctocogene roxaparvovec increased FVIII activity from baseline and improved haemostasis compared with FVIII prophylaxis for most participants. The 6 × 10¹³ vg/kg dose was more efficacious than 4 × 10¹³ vg/kg. No concerning long-term safety signals were identified.
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Raheja et al. (2026) studied this question.
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