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Ceprotin, a concentrated form of protein C made from the plasma of healthy human blood donors, was recently licensed as an orphan drug by the FDA. Severe congenital protein C deficiency is a rare genetic defect found in 1 to 2 newborns for every 1 million births. Patients with insufficient levels of protein C experience abnormally high numbers of blood clots.
Ceprotin's approval was based on data from a pivotal multicenter, open-label, nonrandomized phase 2/3 study that evaluated the safety and efficacy of Ceprotin in patients with severe congenital protein C deficiency for the (on-demand) treatment of acute thrombotic episodes, such as purpura fulminans (PF), warfarin-induced skin necrosis (WISN), and other thromboembolic events, and for short-term or long-term prophylaxis. The study included 18 patients ranging in age from newborn to 25.7 years. Ceprotin was demonstrated to be effective in 94% of the episodes of PF. In the remaining 6% of patients, the treatment was found ?effective with complications? because they required a dosage adjustment. Inadequate data were available for the treatment of WISN.
Currently, patients must take oral or injectable anticoagulants to prevent blood clots. The new product is not intended to replace these agents. Ceprotin is intended to treat these patients when they are faced with a life-threatening situation from venous thromboembolism or PF.