Eptacog Beta Effective for Severe Bleeds in Hemophilia With Inhibitors
A post hoc analysis of the phase 3 PERSEPT 1 and PERSEPT 2 trials has demonstrated that eptacog beta—a recombinant activated human factor VII (rFVIIa)—can effectively and safely manage severe bleeding episodes (BEs) in individuals with hemophilia A or B and inhibitors (PwHABIs). The study highlights eptacog beta’s potential to provide hemostatic control in complex bleeding situations where rapid intervention is critical.
Across both trials, seven PwHABIs aged 1 to 50 years treated 8 severe or life-threatening BEs with eptacog beta. Treatment regimens involved initial doses of 225 μg/kg (or 75 μg/kg depending on the randomized dosing schedule), followed by 75 μg/kg infusions at predetermined intervals. Hemostasis was achieved in 7 of the 8 cases, including 3 intracranial hemorrhages (ICH), within durations ranging from 25 minutes to 96 hours.
These results are consistent with previous findings involving eptacog alfa, another rFVIIa product, though eptacog beta may offer advantages in pharmacokinetics. Importantly, no thrombotic events were reported in any of the 96 administered infusions, and the treatment was well tolerated.
While one patient’s bleeding episode did not respond adequately to eptacog beta, deviation from the dosing protocol and at-home administration may have contributed. Additionally, the limited number of cases and inconsistent adherence to the protocol underscore the need for further research.
Overall, this analysis supports eptacog beta as an effective bypassing agent for managing severe BEs in PwHABIs. The 225 μg/kg regimen is approved by the US Food and Drug Administration and European Medicines Agency, and it appears especially useful in high-stakes clinical settings. Further evaluation is underway in the phase 4 ATHN-16 trial.
Reference
Young G, Mahlangu J, Boggio LN, et al. Treatment of severe bleeds with eptacog beta in hemophilia A or B with inhibitors: a post hoc analysis of the PERSEPT 1 and 2 trials. Blood Vessel Thromb Hemost. 2025;2(3):100047. doi:https://doi.org/10.1016/j.bvth.2025.100069
