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FDA Approves New Once-weekly FVIII Therapy

Hemophilia A (HemA) is a rare blood disorder caused by deficient or dysfunctional Factor VIII (FVIII), leading to impaired clotting and spontaneous joint bleeds.

The FDA has recently approved a new treatment for HemA, Altuviiio, which joins an established market of long-acting FVIII replacement therapies. Approval was granted based on the XTEND-1 trial, which achieved an annualized bleed rate of 0 and a 100% resolution of target joint bleeds. For individuals who did experience bleeds, 97% of bleeding episodes were resolved with one infusion.

Differentiating itself from others in the class, once-weekly Altuviiio is the first FVIII therapy shown to break through the von Willebrand Factor ceiling, which has historically limited the half-life of other products. This allows Altuviiio to maintain FVIII levels >40% for the majority of the week, with levels remaining at 15% by day 7 – providing patients with lasting and measurable protection.

Adding to the excitement, the FDA is set to release their decision for the first HemA gene therapy by end of March and a novel HemA/B siRNA therapy is currently under clinical review. We’ll be sure to keep our eye on the hemophilia market this year!

Written by Rhiannon


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