The US Food and Drug Administration (FDA) has approved CSL Behring’s Corifact, the first product intended to prevent bleeding in people with congenital Factor XIII deficiency, a rare genetic defect.
The FDA cleared Corifact based on results from a clinical study involving 14 people with the condition, including children.
Corifact has been awarded orphan-drug designation as it is intended to treat a rare disease or condition, and has been approved for marketing under the FDA’s accelerated approval regulations.
Corifact is made from the pooled plasma of healthy donors.
Factor XIII deficiency is rare genetic defect affecting one in three-five million people in the US.
Patients with congenital Factor XIII deficiency do not make enough Factor XIII, a substance in the blood that is important for normal clotting, and people with this condition are at risk for life-threatening bleeding.