FDA approves product to prevent bleeding in people with rare genetic defect

The U.S. Food and Drug Administration last week approved Corifact, the first product intended to prevent bleeding in people with the rare genetic defect congenital Factor XIII deficiency.

Patients with congenital Factor XIII deficiency don’t make enough Factor XIII, a substance that circulates in the blood and is important for normal clotting. Without treatment, people with the condition are at risk for life-threatening bleeding.

Congenital Factor XIII deficiency is rare and affects 1 out of every 3 million to 5 million people in the United States. 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,” said Karen Midthun, M.D., director of the FDA’s Center for Biologics Evaluation and Research.

Corifact received orphan-drug designation by the FDA because it is intended for use in a rare disease or condition. It was approved for marketing under the FDA's accelerated approval regulations that require an on-going study to demonstrate that patients actually receive the clinical benefit predicted by the data obtained so far.

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.

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FDA Approves First Additive Solution for Platelet Storage Up to 5 Days

/PRNewswire/ -- The U.S. Food and Drug Administration today approved InterSol, the first solution approved to replace a portion of plasma used when storing platelets for up to five days, allowing some of the donor plasma to be used for other purposes.

In the United States, platelets, which are blood components that help with blood clotting, have been stored in donor plasma. About 65% of that plasma is replaced with the InterSol platelet additive solution.

Platelets are used to prevent or treat bleeding in individuals undergoing chemotherapy for cancer, after major trauma, during or after surgery, and in individuals who do not produce platelets. InterSol solution does not have a therapeutic effect by itself but acts to provide nutrients and an appropriate environment in which to store platelets.

"The approval of InterSol is a significant step in the development and marketing of novel processes for the storage of platelets," said Karen Midthun, MD, acting director of FDA's Center for Biologics Evaluation and Research.

The InterSol platelet additive solution is an aqueous solution of various salt components. It replaces a proportion of plasma in the storage of platelets collected by an automated instrument.

InterSol solution is manufactured by Fenwal Inc., Lake Zurich, IL.

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