Altuviiio bests others as hemophilia A factor replacement therapy
Once-weekly treatment results in fewer bleeding episodes, study finds
For adults and adolescents with hemophilia A without inhibitors, Altuviiio (efanesoctocog alfa) may work better than other standard or extended half-life factor replacement therapies at preventing bleeding episodes, while requiring less frequent injections, a study found.
Researchers made an indirect comparison of data from XTEND-1 (NCT04161495) and published data for other approved factor replacement therapies, and found that once-weekly prophylaxis with Altuviiio resulted in fewer bleeding episodes, including those occurring spontaneously and into the joints.
The study, “Efanesoctocog Alfa versus Standard and Extended Half-Life Factor VIII Prophylaxis in Adolescent and Adult Patients with Haemophilia A without Inhibitors,” was published in Advances in Therapy. It was funded by Sanofi, which markets Altuviiio in the U.S., and Sobi, which markets the therapy as Altuvoct in Europe and other markets.
Hemophilia A is caused by mutations in the gene that provides instructions for producing factor VIII (FVIII), a clotting protein found in the blood. When FVIII is faulty or missing, the blood cannot clot well. As a result, patients experience frequent episodes of excessive bleeding.
Factor replacement therapy, which supplies the body with a working version of the clotting protein that is faulty or missing, can prevent or reduce the frequency of bleeding episodes in these patients. Currently available options usually require two to four injections a week, but Altuviiio works with a once-weekly injection, making treatment easier and less burdensome.
Longer-lasting formula
Like many other factor replacement therapies, Altuviiio contains a lab-made version of FVIII that’s fused with another clotting protein called von Willebrand factor that helps protect it from breaking down. Altuviiio also contains an Fc antibody domain to recycle FVIII back into the bloodstream and XTEN proteins to make it last longer, reducing the need for frequent injections.
In XTEND-1, an open-label, Phase 3 study enrolling more than 150 adults and adolescents with severe hemophilia A, ages 12 and older, prophylaxis or preventive treatment with Altuviiio resulted in fewer bleeding episodes compared with a period before the study when patients were receiving another factor replacement therapy.
“While existing data support the use of [Altuviiio] as an effective option in haemophilia A, there are currently no head-to-head clinical trials directly comparing the efficacy of existing prophylaxis therapies,” the researchers wrote.
To make an indirect comparison of Altuviiio and other available replacement therapies, researchers collected data from multiple Phase 3 studies and adjusted them to account for differences in patient characteristics. These included trials testing two different standard half-life factor replacement therapies: Advate (octocog alfa) and Kovaltry (octocog alfa), and four extended half-life factor replacement therapies: Adynovate (rurioctocog alfa pegol), Eloctate (efmoroctocog alfa), Esperoct (turoctocog alfa pegol), and Jivi (damoctocog alfa pegol).
Altuviiio performed better than other extended half-life factor replacement therapies, reducing the annual bleeding rate — a measure of the number of bleeds adjusted to a one-year time window — for any bleeds by a mean of 2.24 bleeds per year, spontaneous bleeds by 1.52, and joint bleeds by 1.60.
When compared with standard half-life factor replacement therapies, Altuviiio was even more effective. It reduced the annual bleeding rate for any bleeds by a mean of 3.61 bleeds per year, spontaneous bleeds by 2.52, and joint bleeds by 3.42.
Compared with standard or other extended half-life factor replacement therapies, “once-weekly [Altuviiio] therapy reduced the rates of any bleeds, including spontaneous (that happen for no apparent reason) and joint bleeds,” the researchers wrote.
This means that “as well as reducing the number of injections needed per week, [Altuviiio] may work better at preventing bleeds than current [standard] or [extended half-life] FVIII replacement therapies,” which “could have a large impact on the lives of people with severe haemophilia A,” they wrote.
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