FDA Approves Corifact for Treatment of Rare Type of Hemophilia
The U.S. Food and Drug Administration (FDA) approved the drug Corifact this week, giving it an orphan-drug designation because it is intended for use in a rare disease or condition.
The rare condition Corifact will be used to treat is the rarest form of hemophilia caused by a factor deficiency, Factor XIII deficiency.
Factor XIII deficiency is rare congenital genetic defect and affects 1 out of every 3 million to 5 million people in the United States. Patients with this genetic defect don’t make enough Factor XIII, a protein responsible for stabilizing the formation of a blood clot.
Factor XIII deficiency is inherited in an autosomal recessive fashion, affecting both sexes equally. No racial or ethnic group is disproportionately affected.
In the absence of Factor XIII, the blood will clot, but the clot doesn’t maintain it’s stability and is easily dislodged or disintegrate allowing bleeding to continue or recur.
The prolonged bleeding that is associated with Factor XIII is usually associated with trauma. Newborns with Factor XIII deficiency may have umbilical cord bleeding.
With minor trauma, the deficiency may lead to soft tissue bruising or mucosal bleeding. In patients with Factor XIII deficiency who have surgery, bleeding immediately after surgery is usually not excessive, but can be delayed.
Among severe patients there is a high risk of head bleeds with or without trauma. Women who go untreated risk spontaneous abortion or miscarriage.
Common characteristics include soft tissue bleeds, menorrhagia (heavy menstrual periods), joint bleeding, and persistent bleeding during circumsicion or at the site of the umbilical cord.
Diagnosis is made by normal coagulation screening tests and a detailed family history. Specific factor XIII assays can confirm the diagnosis. The condition can also be defined by a clot solubility test.
Corifact is the first product approved in the United States intended to prevent bleeding in people with Factor XIII deficiency.
“This product helps fill an important need,” said Karen Midthun, M.D., director of the FDA’s Center for Biologics Evaluation and Research.
The 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 (hepatic) enzymes.
Corifact is made from the pooled plasma of healthy donors. People receiving Corifact may develop antibodies against Factor XIII that may make the product ineffective. It potentially can cause adverse events from abnormal clotting if doses higher than the labeled dose are given to patients.
Corifact is manufactured by CSL Behring of Marburg, Germany.
FDA Press Release