Margaret Ragni, MD, MPH: Antihemophilic Factor VIII Therapy is a Win-Win

Last week the US Food and Drug Administration (FDA) approved once-weekly antihemophilic factor (Recombinant), Fc-VWF-XTEN Fusion Protein-ehtl (ALTUVIIIO™), a first-in-class, high sustained factor VIII replacement therapy for adults and children with hemophilia A.

Previously referred to as efanesoctocog alfa, the therapy is indicated for routine prophylaxis and on-demand treatment to control bleeding episodes, as well as perioperative management for adults and children with hemophilia A. Approval by the FDA was supported by recently published data from the pivotal XTEND-1 phase 3 study.²

In anticipation of the FDA decision, Margaret V. Ragni, MD, MPH, Professor of Medicine and Clinical Translational Science, and Medical Director, Hemophilia Center of Western Pennsylvania, as well as member of the American Society of Hematology (ASH), spoke with HCPLive to discuss the specific functions of antihemophilic factor (Recombinant), Fc-VWF-XTEN Fusion Protein-ehtl.

"It's a much reduced burden of treatment with once-a-week intravenous infusion that gets you to a higher level the entire week, above about 15% so that you rarely have breakthrough bleeds, at least based on the trial," she explained. "About 70% had no spontaneous breakthrough bleeds–that's really impressive–and it reduced, after a year's use their pain, and it improved their quality of life."

Aside from showing superior efficacy, Ragni emphasized that the treatment burden that would be reduced to once-a-week with use of the therapy makes it much simpler. But with the multitude of novel approaches to recombinant therapy, she continued to explain how this one fits into that landscape.

"What's sort of interesting is that this is the real factor VIII, and it's replacing the missing factory–it recapitulates every piece of what factor VIII does, every function, where as with some of the novel therapies, what they do is they sort of circumvent the missing factor, and all they do is bind, they have the binding function, but they don't have the hemostatic function," Ragni said.

Regardless of whether or not a patient even has access to these new approaches, depending on the individual patient, their disease state may not put them in an adequate position to try something new, though they're likely to benefit from Fc-VWF-XTEN Fusion Protein-ehtl.

"Overall, this is a win-win," she said. "It improves life for most patients who have severe hemophilia A by huge amount more than what you would have with standard factors."

References:

1. Iapoce C. FDA Approves First-in-Class Factor VIII Therapy for Hemophilia A. HCPLive. February 23, 2023.
2. von Drygalski A, CHowdry P, Kulkarni R, et al. Efanesoctocog Alfa Prophylaxis for Patients with Severe Hemophilia A. N Engl J Med 2023; 388:310-318. DOI: 10.1056/NEJMoa2209226