The Food and Drug Administration (FDA) has approved Sevenfact (coagulation factor VIIa [recombinant]-jncw; LFB Biotechnologies) for the treatment and control of bleeding episodes occurring in patients aged ≥12 years with hemophilia A or B with inhibitors.
Sevenfact is a recombinant analog of human Factor VIIa that is produced by recombinant DNA technology using genetically engineered rabbits. The use of Seventfact is contraindicated in patients with known allergy or hypersensitivity to rabbits or rabbit proteins.
The approval was supported by efficacy and safety data from a clinical study that evaluated 27 patients with hemophilia A or B with inhibitors, which included treatment of 465 mild or moderate and 3 severe bleeding episodes. The efficacy of Sevenfact was assessed 12 hours after the initial dose.
Results showed that overall, 86% of mild or moderate episodes were treated successfully (defined as requiring no further treatment, no administration of blood products and no increase in pain after 12 hours from the initial dose). The proportion of mild or moderate bleeding events with hemostatic efficacy at 12 hours was 82% in the 75mcg/kg dose group and 91% in the 225mcg/kg dose group.
Hemostasis was achieved at 12 hours in the 3 severe bleeding events. One severe bleed was treated with three 225mcg/kg doses administered every 6 hours, which was a deviation from the study protocol-specified dosing. The remaining 2 patients were treated with 1 and 5 doses of Sevenfact, respectively.
With regard to safety, the most common adverse reactions were headache, dizziness, infusion site discomfort, infusion site hematoma, infusion-related reaction and fever. Sevenfact carries a Boxed Warning about an increased risk of serious arterial and venous thrombotic events following administration; patients should be monitored for signs or symptoms of activation of the coagulation system and for thrombosis.
“Today’s approval provides another treatment option for the control of bleeding episodes in adults and adolescents with hemophilia who have developed inhibitors,” said Dr Peter Marks, MD, PhD, director of the FDA’s Center for Biologics Evaluation and Research. “In addition to being an important option for patients, Sevenfact is the first product for hemophilia treatment that contains an active ingredient obtained from rabbits genetically engineered to produce a protein necessary for blood coagulation.”
Sevenfact is supplied as a lyophilized powder in single-use vials (1mg and 5mg per vial) with diluent for reconstitution in a prefilled syringe.
For more information visit fda.gov.