Ceprotin

Generic name:Protein C Concentrate (Human)
Drug class:Miscellaneous coagulation modifiers
Approval date: March 30, 2007
Company: Baxter Healthcare Corporation
Treatment for: Severe Congenital Protein C Deficiency

Medically reviewed by Drugs.com. Last updated on Sep 10, 2021.

Ceprotin approved by FDA

Severe congenital Protein C deficiency results in a hypercoagulable state, meaning there is an abnormal tendency for blood clotting. This can cause severe, often life-threatening blood clots in small blood vessels, which if left untreated could result in blindness, severe brain damage, multi-organ failure and death. The disease manifests in children very early in life, often in utero or in the first few days of life. Incidence of severe congenital Protein C deficiency has been estimated to be one to two for every million births. Currently, there are fewer than 20 known cases of severe congenital Protein C deficiency in the United States. FDA has granted Ceprotin orphan drug status.

Ceprotin is the first FDA approved therapy for patients with severe congenital Protein C deficiency. It is indicated for patients with severe congenital Protein C deficiency for the prevention and treatment of venous thrombosis and purpura fulminans (PF) (a severe skin and systematic blood clotting disorder). Ceprotin is indicated as a replacement therapy for pediatric and adult patients.

Ceprotin Clinical Data

Ceprotin's approval is based on data from a pivotal multi-center, open- label, non-randomized, Phase II/III study, which evaluated the safety and efficacy of Ceprotin in subjects with severe congenital Protein C deficiency for the (on-demand) treatment of acute thrombotic episodes, such as PF, warfarin-induced skin necrosis (WISN), and other thromboembolic events, and for short-term or long-term prophylaxis. The study included 18 patients (nine male and nine female) ranging in age from newborn to 25.7 years. Ceprotin was demonstrated to be effective in 94 percent of the episodes of PF. In the remaining six percent of patients, the treatment was found "effective with complications" because they required a dosage adjustment. Inadequate data were available for the treatment of WISN.

When compared with the efficacy ratings for 21 episodes of PF (historical control group), subjects with severe congenital Protein C deficiency were more effectively treated with Ceprotin than those treated with modalities such as fresh frozen plasma or conventional anticoagulants. Ceprotin also demonstrated effectiveness in reducing the size and number of skin lesions in patients. Treatment with Ceprotin healed non-necrotic skin lesions after a median of four days, and necrotic skin lesions were healed after a median of 11 days.

Seven patients took Ceprotin as a preventive measure before surgery or anticoagulation therapy and had no associated blood clotting complications. Eight patients who were given Ceprotin as a long term preventive measure, again did not experience any blood clotting compli...