Bypassing agent Sevenfact reduces bleeds in patients with inhibitors
Treatment effective in patients who'd failed to respond to other therapies
Sevenfact (eptacog beta) was deemed safe and effective at stopping bleeds in patients with hemophilia A or B with inhibitors, according to real-world data from a case series study in the U.S.
The treatment was also more affordable than other similar therapies and effective even in patients who’d failed to respond to other therapies.
The study, “Real-world effectiveness of eptacog beta in patients with haemophilia and inhibitors: A multi-institutional case series,” was published in Haemophilia.
People with hemophilia A and B lack specific blood clotting factors, proteins that help form blood clots to prevent excessive bleeding. Factor VIII (FVIII) is missing or is defective in people with hemophilia A, while factor IX (FIX) is missing or dysfunctional in hemophilia B.
Replacement therapy, a standard of care for both types of hemophilia, provides a version of the missing clotting factor to prevent or reduce the frequency of bleeds. Regular treatment can lead to neutralizing antibodies, or inhibitors, developing against these clotting factors that may lower their effectiveness, however.
Sevenfact is a bypassing agent that’s designed to promote clotting in hemophilia patients with inhibitors against FVIII or FIX. It’s approved for those ages 12 and older in the U.S.
Sevenfact’s safety, effectiveness
Here, a team led by researchers at the University of North Carolina School of Medicine reviewed data from 14 patients treated with Sevenfact at seven centers in the U.S. to better understand its real-world safety and effectiveness. Eleven patients had hemophilia A and three had hemophilia B. Sevenfact was administered in most cases as a prophylactic treatment to prevent bleeds or to manage breakthrough bleeds, and more rarely to control bleeding during surgery.
The bypassing agent also was administered where patients showed a poor response to NovoSeven RT (eptacog alfa), an approved bypassing agent marketed by Novo Nordisk.
Sevenfact was effective in 96% of the cases at controlling bleeds and resolving symptoms when used as a first-line therapy, results showed. Its effectiveness was maintained across a broad range of therapeutic doses, which makes it suitable to adjust according to patient’s needs.
In eight cases where patients had inadequate control or lack of response to NovoSeven RT, treatment with Sevenfact led to a complete resolution of most bleeds.
In two patients who continued having bleeds despite treatment with NovoSeven RT, prophylactic treatment with Sevenfact significantly reduced the bleed frequency. Sevenfact also reduced dosing frequency and infusion volume, without impacting treatment efficacy, in one patient who was receiving prophylactic treatment with FEIBA (activated prothrombin complex concentrate), another bypassing agent.
Sevenfact also provided effective bleed control for three patients who underwent minor surgical procedures. It was also estimated to be 46%-72% more cost-effective than NovoSeven RT. No safety concerns or adverse events were reported.
“In this case series, [Sevenfact] was safe, effective, and economical as first-line therapy, treatment of refractory [bleeding events], management of perioperative bleeding, or prophylaxis in hemophilia patients with inhibitors,” the researchers wrote.
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