FDA Approves First Biologic to Treat Rare Clotting Disorder

The FDA has licensed Baxter Healthcare’s (Deerfield, IL, www.baxter.com) Ceprotin, the first biologic treatment for patients with a rare genetic defect that can cause a potentially life-threatening clotting disorder. Ceprotin is a concentrated form of Protein C, a substance normally manufactured in the liver that circulates in the plasma in very small amounts.

Severe congenital Protein C deficiency is a rare genetic defect found in one to two newborns for every million births. This protein plays an important role in controlling blood coagulation by preventing the formation and growth of blood clots. Patients with insufficient levels of Protein C experience abnormally high numbers of blood clots. Patients with severe inherited Protein C deficiency must take oral or injected anticoagulant drugs on a regular basis to avoid blood clots.

Complete absence of Protein C can be fatal. Ceprotin is intended to treat high-risk 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.

FDA has granted Ceprotin orphan drug status. This designation provides a manufacturer with financial incentives to develop a drug or biologic to treat a rare disease (affecting fewer than 200,000 people in the US). Since 1983, more than 200 drugs and biological products have been brought to market in this way.