Last week, FDA approved Advate, a hemophilia A treatment containing no human blood components. The drug will be available in pharmacies within two to five weeks. Baxter Healthcare Corp., the drug's developer, has not determined its average wholesale price, although it expects Advate to cost slightly more than other hemophilia treatments.
Hemophilia A, the most common form of hemophilia, is caused by a deficiency in or the inactivity of the blood-clotting protein factor VIII. Previously, most hemophilia treatments used factor VIII -- either genetically engineered or from plasma -- administered by intravenous infusion to stop bleeding episodes or to keep patients on a preventive regimen. The danger of using factor VIII from human blood donors is that one donor can contaminate the whole pool. This happened in the 1980s, according to Dr. Steven Pipe of the hemophilia program at the University of Michigan, when 80 percent of patients who received transfusions contracted HIV or hepatitis C.
Although genetically engineered treatment has not, in practice, acted as a carrier for viruses or diseases, Pipe noted the potential that some viruses might not be tested for or cleared by the purification technique.
Advate, unlike previous genetically engineered treatments, does not use the blood protein albumin as a stablilizer, but rather a combination of sugars, amino acids and salts to stabilize factor VIII. "This will now completely eliminate the nagging risk for transfusion for any blood-borne infectious agents," Pipe said.