Ceprotin was shown to be "effective" for 94 percent of the participants in a trial study. Only six percent of the patients studied had complications due to dosage adjustment needs, yet these patients still experienced effective results.
Eighty percent of the study participants had "excellent" results from treatments with Ceprotin for vein blood clots and 20 percent had "good" results.
Although the rare disease only affects fewer than 200,000 people in the U.S., it is a severe congenital protein C deficiency found in one or two newborns from every million births.
"This product offers much-needed treatment for the small number of patients with severe inherited protein C deficiency," according to Dr. Jesse Goodman, who is the FDA's Center for Biologics Evaluation and Research Director.
"If left untreated, clotting may result in blindness, severe brain damage, multi-organ failure and death for these patients," adds Dr. Goodman.
Patients who suffer with the severe form of this disorder must be treated on a regular basis to avoid blood clots.
Ceprotin was found to play an important role for both prevention and treatment. The drug acts to replace a substance normally activated in the body. It is formulated by using the plasma from healthy blood donors.
