FDA Approves First Treatment for Genetic Clotting Disorder

The FDA has approved Baxter's Ceprotin, the first treatment for patients with a rare genetic blood clotting disorder.

Ceprotin, made from donated human blood, can treat patients with life-threatening clots in their veins, as well as patients with a skin and systemic clotting disorder called purpura fulminans.

Patients with the disorder are prone to high numbers of clots because of a deficiency of Protein C, which controls blood coagulation. Ceprotin is the first biologic treatment for patients with the defect, which is found in one to two of every 1 million newborns, the FDA said. Blood clots can cause blindness, brain damage, organ failure and death.

In a clinical study, Ceprotin rated "excellent" in treating 80 percent of blood clots in the veins, and "good" in the other 20 percent, according to the FDA. The drug was also effective in treating all of the purpura fulminans patients.

The FDA previously granted Ceprotin orphan drug status, which gives manufacturers financial incentives to develop products that treat rare diseases.

In clinical trials, the most common adverse reactions were rashes, itching and lightheadedness, the agency said.