The FDA ( Food and Drug Administration ) has granted marketing approval for Corifact, Factor XIII Concentrate ( human ), for the routine prophylactic treatment of congenital factor XIII ( FXIII ) deficiency.
Corifact, already available for use in 12 countries throughout the world under the trade name Fibrogammin- P, is the first and only FXIII concentrate approved in the United States.

Congenital FXIII deficiency, also known as fibrin-stabilizing factor deficiency, is a rare and potentially life-threatening bleeding disorder in which blood clots normally, but the clots formed are unstable, leading to recurrent bleeding. It is estimated that the condition affects one person in two million, with an incidence in the U.S. of approximately 150 people.

Symptoms of congenital FXIII deficiency include bleeding from the umbilical cord after birth, poor wound healing, miscarriages, subcutaneous bleeding, and excessive bleeding in joints and muscles following trauma. Patients lacking the FXIII protein are also at high-risk for intracranial hemorrhage ( ICH ), bleeding inside the skull that can be life-threatening. Studies have shown that between 25 and 60% of FXIII-deficient patients will experience an ICH at least once during their lifetime.
For a doctor to accurately diagnose FXIII deficiency, specific blood testing and detailed family history are necessary.

Corifact, an intravenous ( IV ) infusion given approximately every 28 days, is indicated for routine prophylactic treatment of congenital FXIII deficiency. There are no controlled trials demonstrating a direct benefit on treatment of bleeding episodes with Corifact.
Corifact is a fibrin-stabilizing factor concentrate that provides both A- and B-subunits to protect against FXIII deficiency. Clinical studies have shown that Corifact immediately increases FXIII levels in the blood. The concentrated formula is dosed, depending on the patient's weight, to maintain trough FXIII levels between 5 and 20 percent of normal.

Corifact is packaged as lyophilized powder in a single-use vial with high stability and a long shelf life. The concentrate is stable for up to six months at room temperature ( 25 degrees C ) and 24 months when refrigerated ( 2-8 degrees C ). Corifact also has a low infusion volume, which could help reduce administration time.

Corifact is also available as Fibrogammin- P in Argentina, Austria, Belgium, Brazil, France, Germany, Great Britain, Indonesia, Israel, Japan, Luxembourg, and Switzerland.

Corifact is not for individuals who experience symptoms of hypersensitivity with known anaphylactic or severe systemic reactions to human plasma-derived products or any components in the formulation. In clinical studies, the most common adverse reactions reported following treatment with Corifact include hypersensitivity ( including allergy, rash, pruritus, erythema ), chills/rise in temperature, arthralgia, headache, elevated thrombin-antithrombin levels, and an increase in hepatic enzymes.
Physicians should monitor a patient's trough FXIII activity level during treatment.
If the expected FXIII level is not attained, or breakthrough bleeding occurs, an investigation to determine the presence of FXIII inhibitor antibodies should be performed.

Corifact undergoes virus inactivation and removal for safety assurance. Corifact is derived from human plasma. The risk of transmission of infectious agents, including viruses and, theoretically, the Creutzfeldt-Jakob disease ( CJD ) agent, cannot be completely eliminated.

Source: FDA, 2011

XagenaMedicine2011