FDA Approves First Treatment for Congenital Factor XIII Deficiency

Kate Johnson

February 23, 2011

February 23, 2011 — The first product intended to prevent bleeding in people with congenital factor XIII deficiency has been approved by the US Food and Drug Administration (FDA).

Corifact, manufactured by CSL Behring, received orphan drug designation because it is intended for a rare condition.

Factor XIII deficiency, also known as fibrin-stabilizing factor deficiency, is a genetic defect that affects 1 of every 3 to 5 million people in the United States.

Without treatment, those with factor XIII deficiency are at risk for life-threatening bleeds. Potential symptoms include soft tissue bruising, mucosal bleeding, and fatal intracranial bleeding. Newborns with the condition may have umbilical cord bleeding.

The new drug is made from pooled plasma of healthy donors and was approved based on the results of a clinical study of 14 people, including children, through the FDA's accelerated approval process.

Regulations for accelerated approval require an ongoing study to demonstrate the predicted clinical benefit in patients.

The most common adverse effects observed were hypersensitivity reactions (allergy, rash, pruritus, and erythema), chills, fever, arthralgia, headache, elevated thrombin-antithrombin levels, and an increase in hepatic enzymes.

The treatment can potentially cause abnormal clotting if doses higher than recommended are given, and some people may develop antibodies to the treatment, rendering it ineffective.

"This product helps fill an important need," said Karen Midthun, MD, director of the FDA's Center for Biologics Evaluation and Research, in a press release.

Since Congress passed the Orphan Drug Act (ODA) in 1983, more than 250 orphan drugs have been developed compared with just 10 such products developed without government assistance before the act, according to the FDA.

The ODA provides financial incentives to drug and biologics manufacturers to focus on treatments for orphan diseases, defined as conditions that affect fewer than 200,000 people nationwide.

"As a result of the ODA, treatments are available to people with rare diseases who once had no hope for survival," says the FDA Web site.

Corifact is already available for use in 12 countries under the trade name Fibrogammin-P, according to the manufacturer's press release.

The FDA approval notice can be found on the agency's Web site.

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