Newly published data from a phase 3 clinical study suggests that the recombinant factor VIIa bypassing agent (BPA) eptacog beta was used to safely and effectively treat and control bleeding in children with hemophilia A or B with inhibitors, 12 years of age or younger.
Eptacog beta is unique in that its active ingredient, a recombinant analog of the human FVII protein, is generated in the mammary gland of genetically engineered rabbits. It’s current indication – approved by the FDA in 2020 under the brand name SEVENFACT® – is limited to the treatment and control of bleeding episodes occurring in adults and adolescents (12 years of age and older) with hemophilia A or B with inhibitors.
The phase 3 study, known as PERSEPT 2 is an international multicenter trial. Males with hemophilia A or B and inhibitor under the age of 12 were eligible. In all, 25 enrolled participants initially received infused doses of eptacog beta at either 75 or 225 micrograms of medication per kilogram (ug/kg) of body weight, proceeded by follow-up infusions at the 75 mcg/kg dose at predetermined intervals with adjustments made based on clinical responses.
Treatment of mild or moderate bleeding episodes (BEs) were deemed successful when each patient met the following criteria – 1) A hemostasis evaluation of ‘excellent’ or ‘good’ was obtained; 2) no additional infusions of eptacog beta were administered within 24 hours after the first ‘excellent’ or ‘good’ response was noted; 3) no alternative hemostatic agent or blood product was needed; 4) pain associated with the BE did not increase following the initial ‘excellent’ or ‘good’ response.
Based on these criteria, investigators reported proportional treatment success. In the 25 participants who had collectively experienced 546 mild or moderate BEs, success was achieved in 65% in the 75 μg/kg initial dose regimen (IDR) and 60% in the 225 μg/kg IDR 12 hours following the initial infusion.
Treatment success proportions rose significantly at the 24-hour mark, to 97% for the 75 μg/kg IDR and 98% for the 225 μg/kg IDR, respectively. In addition, there were no reports of thrombotic events, allergic reactions, antibody response, or treatment-related adverse events.
Investigators concluded that “a significant proportion of BEs were successfully treated at 12 hours; and nearly all BEs were resolved at 24 hours. As such, eptacog beta potentially offers an important therapeutic option to patients, caregivers, and health care providers for BE treatment.”
These findings were published by the journal Haemophilia online ahead of print on April 27th – Read the full open access article.