Last month, the U.S. Food and Drug Administration (FDA) approved Corifact™, a plasma-derived factor XIII (FXIII) product, for the routine prophylactic treatment of congenital FXIII deficiency. The FDA approval was made after positive results from a clinical study of 14 patients with congenital FXIII deficiency.
FXIII deficiency is a rare disease that affects 1 out of every 3 to 5 million people in the U.S., or approximately 150 people. The condition is characterized by blood that clots normally, but the clots are unstable, so bleeding recurs. FXIII deficiency can cause umbilical cord bleeding in some newborns, soft tissue bruising, mucosal bleeding and potentially fatal intracranial hemorrhage (ICH). Studies have shown that 25% to 60% of people with FXIII deficiency experience at least one ICH during their lifetime.
Corifact™, the only FXIII concentrate approved in the U.S., received orphan drug designation from the FDA’s Office of Orphan Products Development. This allows for accelerated approval so that drugs or biologics become available to patients with rare, life-threatening diseases who need them. More than 1,400 drugs and biologics have been designated as orphan drugs. Since 1983, more than 250 of them have been approved for marketing.
“This product helps fill an important need,” said Karen Midthun, MD, director of the FDA’s Center for Biologics Evaluation and Research. Prior to the drug’s approval, patients with FXIII deficiency were treated with cryoprecipitate or fresh frozen plasma. Corifact is given intravenously every 28 days. The most common side effects are allergies and rashes, chills, fever, joint pain, headache and elevated liver enzymes.
According to a CSL press release, Corifact™ is already available for use in 12 countries worldwide under the trade name Fibrogammin®-P.
Source: FDA news release date February 17, 2011, and CSL Behring news release dated February 18, 2011