Brain Damage Newsroom

FDA Approves First Biologic to Treat Rare Clotting Disorder

The U.S. Food and Drug Administration -FDA- licensed Ceprotin, the first biologic treatment for patients with a rare genetic defect that can cause a potentially life-threatening clotting disorder.

Ceprotin is made from the plasma of healthy human blood donors. It is a concentrated form of Protein C, a substance normally manufactured in the liver that circulates in the plasma in very small amounts. Protein C plays an important role in controlling blood coagulation by preventing the formation and growth of blood clots.

Severe congenital Protein C deficiency is a rare genetic defect found in one to two newborns for every million births. Patients with insufficient levels of Protein C experience abnormally high numbers of blood clots. Complete absence of the protein is fatal. Symptoms typically appear soon after birth. Clotting may occur in the blood vessels of the skin, eyes, brain, kidneys and throughout the body.

"This product offers much-needed treatment for the small number of patients with severe inherited Protein C deficiency," said Jesse Goodman, M.D., M.P.H., 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."

Patients with severe inherited Protein C deficiency must take oral or injected anticoagulant drugs on a regular basis to avoid blood clots. Ceprotin is intended to treat these patients when they are faced with a life-threatening situation from blood clots in the veins, or a severe skin and systemic blood clotting disorder known as Purpura fulminans.