MONDAY, April 2 (HealthDay News) -- The U.S. Food and Drug Administration has licensed a new drug, Ceprotin, to treat patients with severe, inherited protein C deficiency. Ceprotin can be used in life-threatening events in patients with the rare disorder, including venous thromboembolism or Purpura fulminans.
The drug, which is derived from human plasma and manufactured by Baxter Healthcare Corp. of Deerfield, Ill., was granted orphan drug status by the FDA, because it is used to treat a disease that affects less than 200,000 people in the United States. Only one to two infants are born with the disease annually.
Licensing was granted after a pivotal trial in which 80 percent of subjects treated for venous blood clots had excellent results and results for the remaining 20 percent were good. The drug was effective in 94 percent of patients treated for Purpura fulminans, while the remaining 6 percent required dose adjustment due to complications.
"This product offers much-needed treatment for the small number of patients with severe inherited Protein C deficiency. If left untreated, clotting may result in blindness, severe brain damage, multi-organ failure and death for these patients," said Jesse Goodman, M.D., director of FDA's Center for Biologics Evaluation and Research, in a statement.
More Information
