FDA approves Corifact to prevent bleeding in people with rare genetic defect

FDA has approved Corifact (CSL Behring), the first product intended to prevent bleeding in people with the rare genetic defect congenital Factor XIII deficiency.

Congenital Factor XIII deficiency affects 1 out of every 3 million to 5 million people in the United States. The deficiency may lead to soft-tissue bruising, mucosal bleeding, and fatal intracranial bleeding. Newborns with Factor XIII deficiency may have umbilical cord bleeding.

“Patients suffering from Factor XIII deficiency have a range of symptom severities and those in emerging markets tend to have fewer options,” Sarah Terry, president, global managing directorat Life Science Analytics, a Datamonitor company, told Formulary.

There have been only slight improvements in treatment options over the years and are no curative therapies on the horizon, added Terry. “Newer products for Factor XIII deficiency focus on the use of second-hand protein-engineering technologies used primarily to improve potency and duration of activity,” she said. “However, the cost of management is still a significant challenge for many patients. And, with any new product, it will be important to see if the cost of treatment is reduced so that greater patient access could ensue.”

FDA approved Corifact based on results of a clinical study of 14 people, including children, with congenital Factor XIII deficiency. The most common side effects observed were hypersensitivity reactions (allergy, rash, pruritus, and erythema), chills, fever, arthralgia, headache, elevated thrombin-antithrombin levels, and an increase in liver enzymes.