Baxter International Inc reported positive results from a late-stage study evaluating routine use of its anti-inhibitor coagulant complex to treat hemophilia, a rare genetic blood clotting disorder.
The data will form the basis of a biologics license application to be filed with the U.S. health regulators in the first quarter of 2013.
The Phase 3 clinical trial investigated the efficacy, safety and health-related quality of life benefits of its so-called anti-inhibitor coagulant complex prophylactic treatment given to maintain health and prevent bleeds compared with on-demand treatment. The study included 36 patients with hemophilia A or B and inhibitors over a 12-month period.
There was a 72.5 percent reduction in bleed rates for the prophylactic treatment group, Baxter said.
The most commonly reported negative side effects in the study were hypersensitivity, dizziness, headache, rash, hypotension and hepatitis B surface antibody positive laboratory test result.
The occurrence of a transitory increase in hepatitis B surface antibodies has been seen in certain plasma-derived products and could be attributed to the passive transfer of antibodies following treatment. None of the subjects showed any signs or symptoms of hepatitis B infection, the company said.
People living with hemophilia do not have enough of, or are missing, one of the blood clotting proteins naturally found in blood. People with hemophilia can experience spontaneous, uncontrolled internal bleeding that is potentially fatal.
More than 400,000 people in the world have hemophilia, according to the World Federation of Hemophilia.
The most common forms of the disease are hemophilia A and hemophilia B. People with hemophilia A lack sufficient amounts of clotting factor and those with hemophilia B, the second most common type, have insufficient amounts of clotting factor IX.
Baxter's drug, if approved, would potentially compete with hemophilia drugs being developed by Biogen Idec Inc. In October Biogen said its experimental treatment for patients with hemophilia A controlled bleeding with fewer treatments in a late-stage clinical trial.
The market for hemophilia A treatments is worth about $5 billion, according to Biogen, while the market for hemophilia B treatments is about $1 billion. The drugs, if approved, would also compete with shorter-acting products, including Baxter's market-leading drug Advate.
Biogen filed recently for U.S. approval for its Factor IX product for hemophilia B. It is on track to file for approval of its Factor VIII product for patients with hemophilia A in the first half of 2013.
(Reporting By Debra Sherman and Toni Clarke; Editing by Theodore d'Afflisio)