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Factor It In: Gene Therapy for Hemophilia A


BioMarin’s Valoctocogene roxaparvovec (ROCTAVIAN™) is poised to be the first gene therapy available in the US for severe hemophilia A. With an anticipated regulatory decision in March 2023, ROCTAVIAN™ could transform the treatment landscape for hemophilia A.


Hemophilia A is a rare genetic disorder characterized by missing or defective clotting factor VIII (FVIII), leading to an increased risk of bleeding. Those with the most serious form of hemophilia experience spontaneous bleeding events, which may be life-threatening depending on the location.


In contrast to common prophylactic regimens which require regular, life-long infusions/injections, ROCTAVIAN™ is administered to the patient as a one-time infusion and enables the production of an individual’s missing FVIII.


ROCTAVIAN™ met all primary and secondary efficacy end points in the 3-year analysis of the largest and longest Phase 3 gene therapy study for hemophilia A. The results demonstrated an 80% reduction in the mean annualized bleed rate from baseline. Further, 92% of patients remained off prophylaxis at the end of the 3-year period.


If approved, this therapy could serve as a new, durable treatment option for patients with hemophilia A.


Written by Zaraa Malvat

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