Several new blood clotting agents are being developed to reduce the number of regular intravenous infusions needed by hemophilia patients to prevent traumatic bleeding.
"It was amazing actually," said hemophilia patient Travis Roop, who was able to receive infusions of a blood clotting agent every two weeks. The change meant he could jog for the first time.
"It was a life-changing experience," he said. "I always avoided trauma to my left ankle... With this, I knew it would take care of itself."
Those who have hemophilia have a defect in a gene that regulates the body's production of proteins called clotting factors. This can lead to spontaneous bleeding and severe bleeding after injuries and surgery.
In order to avoid joint damage and other complications, patients who have severe forms of hemophilia require regular infusions of relatively short acting and expensive clotting factors. Number of infusions and volume varies with the patient's medical status and body weight.
Hemophilia mostly affects men. It has been called the "Royal Disease" because Britain's Queen Victoria had it and because many of the ruling families of Europe were affected by it.
Some experts say that new treatments for hemophilia could help reduce the price of existing hemophilia products, which can reach $300,000 or more for one patient. The U.S. Food and Drug Administration is deciding whether to approve Alprolix, a new long-lasting hemophilia B clotting factor from Biogen Idec and partner Swedish Orphan Biovitrum. Novo Nordisk of Denmark expects to file for regulatory approval of the long-acting hemophilia B drug N9-GP next year.
If the makers of the new hemophilia agents decide to price their product at the same level as existing shorter-acting products, patients will have a relatively easy time deciding to try the new factors, said Dr. Guy Young, director of the Hemostasis and Thrombosis Center at Children's Hospital Los Angeles.
"If they come in at a similar annual price, then companies that are making recombinant drugs will be forced to drop their price," Young said.
About one in 5,000 men is born with hemophilia A and one in 25,000 men is born with hemophilia B worldwide each year.
In 2011 the global market for hemophilia drugs totaled $8.5 billion. According to Morningstar, the market has the potential to grow to $11.4 billion by 2016.
The new hemophilia B products can be administered every 10 days or two weeks, compared with the current practice of giving infusions to patients, especially children, every two to three days.
"Patients don't always get the doses they are supposed to get," said Dr. Marion Koerper, medical director of the National Hemophilia Foundation and professor of hematology at the University of California at San Francisco Children's Hospital.
Young said he predicts most hemophilia B patients will end up taking the new products.
