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 multicen-ter, 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 throm- botic 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.
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