In March 2007, the U.S. Food and Drug Administration (FDA) granted a license for Ceprotin, a plasma-based therapy for people with severe congenital protein C deficiency, an inherited clotting disorder. The disorder is very rare, occurring in only one to two newborns for every million births. Protein C, which is produced in the liver, is released into the plasma to control clot formation. A defect in the gene responsible for its production results in increased susceptibility to potentially dangerous blood clots.
Patients with severe inherited protein C deficiency typically take regular doses of oral or injected anticoagulants to prevent clots. Ceprotin, manufactured by Baxter Healthcare Corporation, is for use by patients for prevention and treatment of acute, life-threatening blood clots in the veins or purpura fulminans, a severe skin and systemic blood clotting disorder. In tests, the drug decreased the size and number of skin lesions.
“This product offers much-needed treatment for the small number of patients with severe inherited protein C deficiency,” said Jesse Goodman, MD, MPH, director of the 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.”
The FDA had previously granted Ceprotin “orphan drug” status, a designation for promising pharmaceuticals to treat rare diseases or conditions, such as protein C deficiency. It is estimated that there are fewer than 20 cases of severe congenital protein C deficiency in the United States. Since the FDA’s Office of Orphan Products Development opened in 1983, more than 200 drugs and biological products have been developed.
Source: FDA news release dated March 30, 2007