Baxter's Ceprotin Approved for Clotting Disorder

The FDA announced it has approved Ceprotin, the first biologic treatment for patients with a rare genetic defect that can cause a potentially life-threatening blood 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.

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.

The drugmaker Baxter enrolled all available patients for the drug's pivotal trial. In 94 percent of the patients studied for Purpura fulminans, Ceprotin was found to be effective. In another 6 percent of patients, the treatment was found to be effective with complications.

The seven patients who took Ceprotin as a preventive measure before surgery or anticoagulation therapy experienced no blood clotting complications. Eight patients who were given Ceprotin as a long-term preventive measure did not experience the severe skin and blood clotting events associated with Purpura fulminans.

The FDA had granted Ceprotin orphan drug status and it reviewed the biologics license application under a priority-review schedule.