Our July 2025 Quick Question asked, “Which is a hemophilia ‘rebalancing’ therapeutic?” The question attracted a modest 22 participants, perhaps an indication that rebalancing therapy was only FDA-approved in 2025 and awaits approval in Canada and Europe. Here are your responses…
- Valoctocogene roxaparvovec-rvox (ROCTAVIAN) 3 (14%)
- Efanesoctocog alfa (ALTUVIIIO) 1 (4%)
- Emicizumab (HemLibra) 7 (32%)
- Denecimig (Mim8) 0 (0%)
- Fitusiran (Qfitlia) 11 (50%)
For a review of past and current hemophilia therapy, click “Differential Discussions (hosted by Malissa Norfolk and David Cabral), “Have We Cured Hemophilia?” and “Hemophilia Part 2.” For a comprehensive open-access review article, click our April 25, 2025 entry, “The Choice of Hemophilia Treatments.”
In short, Fitusiran (Qfitlia), answer 5, was correct. Fitusiran employs RNAi to suppress AT activity to a target level between 10% and 35%. AT is a key control protein that modifies thrombin (FII) and FX activities.
Efanesoctocog alfa (ALTUVIIIO) is an EHL BDD rFVIII concentrate that features an Fc receptor site and a VWF fragment that together extend its half-life 3 to 4X native FVIII, reducing prophylactic infusion frequency to once a week.
Valoctocogene roxaparvovec-rvox (ROCTAVIAN) is the name given to the “one and done” gene transfer therapy FDA-approved in 2023 that manages HA (FVIII deficiency) by inducing hepatocyte FVIII production.
Emicizumab (HemLibra) is the bispecific FVIII bypassing activity that was approved worldwide in 2017 and is effective in HA patients with inhibitors.
Denecimig (Mim8) is a second FVIII bypassing therapy currently awaiting approval in the Americas, Australia, and Europe.
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