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FDA approves first treatment for Factor XIII deficiency
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The first product to prevent bleeding in people with congenital Factor XIII deficiency has been approved by the FDA.
The drug, Corifact (CSL Behring), received orphan drug designation and was approved for marketing under the FDA’s accelerated approval regulations. These regulations require an ongoing study to demonstrate clinical benefit in patients.
Approval is based on results of a clinical study of 14 people, including children, with congenital Factor XIII deficiency. The most common side effects were hypersensitivity reactions (allergy, rash, pruritus, and erythema), chills, fever, arthralgia, headache, elevated thrombin-antithrombin levels and an increase in hepatic enzymes.
Congenital Factor XIII deficiency is rare and affects 1 out of every 3 million to 5 million people in the US. The deficiency may lead to soft tissue bruising, mucosal bleeding and fatal intracranial bleeding. Newborns with Factor XIII deficiency may have umbilical cord bleeding.
“This product helps fill an important need,” Karen Midthun, MD, director of the FDA’s Center for Biologics Evaluation and Research, said in a press release.
Corifact is made from the pooled plasma of healthy donors. People receiving Corifact may develop antibodies against Factor XIII that could make the product ineffective. It has the potential to cause adverse events from abnormal clotting if doses higher than the labeled dose are administered, according to the release.
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