FDA Approves Coagulation Factor VIIa (Recombinant)-jncw for Hemophilia

The FDA has approved coagulation factor VIIa (recombinant)-jncw to control bleeding in patients aged 12 years and older with hemophilia A or B with inhibitors.

“[Coagulation factor VIIa (recombinant)-jncw] 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,” Peter Marks, MD, PhD, Director of the FDA’s Center for Biologics Evaluation and Research, said in a press release.

The active ingredient, which can be used as an alternate to human factor VII, is extracted from the milk of genetically engineered rabbits. The treatment is effective because it can bypass the factor VIII or factor IX reactions that occur in patients with inhibitors.

The approval is based on data from a clinical study evaluating coagulation factor VIIa (recombinant)-jncw in 27 patients with hemophilia A or B with inhibitors who had experienced a total of 465 mild or moderate bleeding episodes and three severe bleeding episodes. Approximately 86% of mild or moderate bleeding episodes were treated successfully with both the lower and higher doses (75 mcg/kg and 225 mcg/kg) of coagulation factor VIIa (recombinant)-jncw, and all three severe bleeding episodes were treated successfully with the higher dose. Treatment success was determined as the bleeding episode resolving within 12 hours, without an increase in pain.

The most common adverse effects associated with coagulation factor VIIa (recombinant)-jncw were headache, dizziness, infusion site discomfort, infusion-related reaction, infusion site hematoma, and fever. The product is not intended for patients who are allergic or hypersensitive to rabbits or rabbit proteins. Patients with risk factors for thrombosis may be at increased risk of serious thrombotic events. If symptoms of a thrombotic event or hypersensitivity reaction occur, the FDA recommends discontinuing coagulation factor VIIa (recombinant)-jncw and seeking medical intervention.

Sources: Healio, April 1, 2020; FDA press release, April 1, 2020.