April 01, 2020
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FDA approves Sevenfact for certain adults, adolescents with hemophilia

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The FDA approved coagulation factor VIIa (recombinant)-jncw for the treatment and control of bleeding episodes among certain adults and adolescents with hemophilia.

The approval applies to use of the therapy for those aged 12 years or older who have hemophilia A or B with inhibitors.

Coagulation factor VIIa (recombinant)-jncw — which contains an active ingredient expressed in genetically engineered rabbits — is marketed as Sevenfact (LFB Biotechnologies).

“[This] approval provides another treatment option for the control of bleeding episodes in adults and adolescents with hemophilia who have developed inhibitors,” Peter Marks, MD, PhD, director of the FDA’s Center for Biologics Evaluation and Research, said in a press release. “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.”

An estimated 20,000 people in the United States have hemophilia. On-demand treatment or prophylaxis with products that contain factor VIII or factor IX are used to manage bleeding episodes, but these products often are not effective for individuals who develop inhibitors to factor VIII or factor IX.

In such cases, administration of products that bypass factor VIII and factor IX reactions are used to promote clot formation and control bleeding.

The active ingredient in Sevenfact is a recombinant analog of human factor VII, expressed in the mammary gland of genetically engineered rabbits and secreted into their milk.

Factor VII is converted into activated Factor VII during purification and processing of the milk.

The FDA’s Center for Veterinary Medicine approved the recombinant DNA construct in the genetically engineered rabbits used for the production of Sevenfact.

“Based on a comprehensive analysis of the scientific evidence, the FDA determined that the recombinant DNA construct inserted in the rabbit’s genomic DNA is safe for the animal and for the people handling the rabbits, and is effective in causing human factor VII protein to be expressed in the rabbits’ milk,” Steven M. Solomon, DVM, MPH, director of the FDA’s Center for Veterinary Medicine, said in the release. “The FDA has also determined under the National Environmental Policy Act that approval of the application will have no significant impact on the environment.”

The FDA based the approval in part on efficacy and safety data from a study that evaluated Sevenfact for 27 patients with hemophilia A or B with inhibitors.

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Study participants received treatment for a combined 465 mild or moderate bleeding episodes, as well as three severe bleeding episodes.

Researchers administered Sevenfact and assessed efficacy of the treatment 12 hours after administration of the initial dose.

Sevenfact — dosed at either 75 mcg/kg or 225 mcg/kg — controlled approximately 86% of mild or moderate bleeding episodes. The higher dose also successfully treated three severe bleeding episodes.

The most common adverse effects associated with Sevenfact included headache, dizziness, infusion site discomfort, infusion-related reaction, infusion site hematoma and fever.

The FDA noted the product is contraindicated for patients with a known allergy to or hypersensitivity to rabbits or rabbit proteins.

In addition, individuals with hemophilia A or B with inhibitors who have other risk factors for thrombosis may be at elevated risk for serious arterial or venous thrombotic events, according to the agency.