The U.S. Food and Drug Administration (FDA) has approved Sevenfact [coagulation factor VIIa (recombinant)-jncw] to treat and control of bleeding episodes that occur in persons 12 years of age and older with hemophilia A or B with inhibitors (neutralizing antibodies).
“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. This approval is an example of our efforts to advance safe biotechnology innovations to support patient health.”
Hemophilia A and hemophilia B are congenital bleeding disorders due to dysfunction or deficiency of Coagulation Factor(F) VIII or IX, respectively.
People with these conditions may bleed for longer periods of time after injury or surgery. They are also susceptible to spontaneous bleeding in muscles, joints and organs, which can be extremely painful and, in some cases, life-threatening. For many patients with these conditions, Factor replacement therapy is effective but in individuals who develop inhibitors to FVIII or FIX, that therapy is no longer an effective option. For those patients, the administration of products such as Sevenfact, which bypass the FVIII and FIX reactions, promotes clot formation and controls bleeding.
In a news release, Peter Marks, MD, PhD, director of the FDA’s Center for Biologics Evaluation and Research said, “Today’s approval provides another treatment option for the control of bleeding episodes in adults and adolescents with hemophilia who have developed inhibitors.”
Dr. Marks added, “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. This approval is an example of our efforts to advance safe biotechnology innovations to support patient health.”
The approval of the drug was largely based on a phase 3 clinical trial of 27 patients with hemophilia A or B with inhibitors. The study, published in Haemophilia found the drug to be effective in providing a clinical response (a composite of objective and pain measures). The most common side effects of Sevenfact were headache, dizziness, infusion site discomfort, infusion related reaction, infusion site hematoma and fever.
Sevenfact is contraindicated in patients with known allergy or hypersensitivity to rabbits or rabbit proteins. The active ingredient of Sevenfact is a recombinant analog of human FVII, that is expressed in the mammary gland of genetically engineered rabbits and secreted into the rabbits’ milk. During purification and processing of the milk, FVII is converted into activated FVII (FVIIa).
Sevenfold was developed by the French company, Laboratoire Francais du Fractionnement et des Biotechnologies S.A. It is currently not known when the medications will be made available for patients in the United States.