Ceprotin Approved by the FDA for Treatment of Severe Protein C Deficiency

The [U.S.] Food and Drug Administration (FDA) has approved the sale of a new drug for the treatment of a rare, inherited clotting disorder. The drug, Ceprotin, is to be used by sufferers of hereditary severe inherited Protein C deficiency. Such individuals must take oral or injected anticoagulant drugs on a regular basis to avoid blood clots.

Protein C is a major physiological anticoagulant. It is a vitamin K-dependent serine protease enzyme that is activated by thrombin into activated protein C (APC). The activated form (with protein S as a cofactor) degrades Factor Va and Factor VIIIa. It should not be confused with C peptide or C-reactive protein or protein kinase C.

The protein C pathway's key enzyme, activated protein C, provides physiologic antithrombotic activity and exhibits both anti-inflammatory and anti-apoptotic activities. Its actions are related to development of thrombosis and ischemic stroke.

Ceprotin, which is manufactured by Baxter Healthcare Corp. (Deerfield, IL, USA), is a concentrated form of Protein C made from the plasma of healthy human blood donors.

The FDA granted Ceprotin orphan drug status, which provided the manufacturer with financial incentives to develop the drug to treat such a rare disease (affecting fewer than 200,000 people in the United States). "This product offers much-needed treatment for the small number of patients with severe inherited Protein C deficiency,” said Dr, Jesse Goodman, director of FDA's Center for Biologics Evaluation and Research. "If left untreated, clotting may result in blindness, severe brain damage, multi-organ failure, and death for these patients.”


Related Links:
Baxter Healthcare Corp
U.S. Food and Drug Administration