FDA Approves Treatment for Adults, Adolescents with Hemophilia A or B and Inhibitors

The FDA has approved coagulation factor VIIA (recombinant)-jncw (Sevenfact, LFB) for the treatment and control of bleeding episodes occurring in adolescents aged 12 years and older with hemophilia A or B with inhibitors.

“In addition to being an important option for patients, [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. This approval is an example of our efforts to advance safe biotechnology innovations to support patient health,” said Peter Marks, MD, PhD, director of the Center for Biologics Evaluation and Research at the FDA, in a prepared statement.

The CDC estimates that there are approximately 20,000 people living with hemophilia in the United States. Patients with hemophilia may bleed for longer time than others after injury or surgery and may also have spontaneous bleeding in muscles, joints, and organs, which may be life-threatening. Individuals with inhibitors may not respond to factor replacement.

The active ingredient of coagulation factor VIIA (recombinant)-jncw is a recombinant analog of human FVII, expressed in the mammary gland of genetically engineered rabbits, and secreted into the rabbit’s milk. During purification and processing the milk, FVII is converted into activated FVII (FVIIa). The recombinant DNA (rDNA) construct in the genetically engineered rabbits used for the production of coagulation factor VIIA was approved by the FDA’s Center for Veterinary Medicine.

A previous clinical study evaluated the safety and efficacy of coagulation factor VIIA (recombinant)-jncw and included 27 patients with hemophilia A or B with inhibitors. This evaluation included 465 mild or moderate, and 3 severe bleeding episodes. The study assessed the efficacy of treatment 12 hours after the initial dose was given. The proportion of mild or moderate bleeding episodes treated successfully both with the lower dose of 75mcg/kg and higher dose of 225 mcg/kg was approximately 86%.

Another study evaluated the safety and pharmacokinetics of 3 escalating doses of coagulation factor VIIA (recombinant)-jncw in 15 patients with severe hemophilia A or B with or without inhibitors. Results from the study were evaluated in the study.

The most common adverse events of coagulation factor VIIA (recombinant)-jncw were headache, dizziness, infusion site discomfort, infusion related reaction, infusion site hematoma, and fever.

REFERENCE

FDA Approves Additional Treatment for Adults and Adolescents with Hemophilia A or B and Inhibitors [news release]. Published April 1, 2020. https://www.fda.gov/news-events/press-announcements/fda-approves-additional-treatment-adults-and-adolescents-hemophilia-or-b-and-inhibitors. Accessed April 2, 2020.