FIX-FIAV

FIX-FIAV is an investigational recombinant Factor IX (FIX) protein variant engineered with four amino acid substitutions (L6F, V181I, K265A, and I383V) that enable it to activate Factor X (FX) independently of its normal cofactor, Factor VIIIa (FVIIIa). This FVIII-independent mechanism of action makes it a novel potential therapeutic for hemophilia A, a disease caused by deficiency or dysfunction of Factor VIII, including in patients who have developed inhibitory antibodies against Factor VIII. Originally developed by researchers at Goethe University Frankfurt and the German Red Cross Blood Donor Service Baden-Württemberg-Hessen, the variant has been licensed by uniQure and studied by Erasmus University Medical Center Rotterdam under the FIVITAS study, a non-interventional, ex vivo/in vitro investigation evaluating whether adding recombinant FIX-FIAV to blood samples from adult men with hemophilia A improves thrombin generation and clotting parameters (e.g., lag time, endogenous thrombin potential, aPTT).

Other names

factor IX-FIAVrecombinant Factor IX-FIAV