FDA Approves New Hemophilia Drug

Approximately 16,000 people suffer from hemophilia A in the United States. The FDA approved a new drug manufactured by Biogen Idec Inc. that controls and prevents bleeding in patients who suffer from this disease.

Two months ago the FDA approved the first new hemophilia treatment since 1997, which was also manufactured by Biogen Idec Inc. That drug, Alprolix, treats hemophilia B and it is based on the same mechanism of action as their newest drug, Eloctate. Now that Biogen Idec has the permission to market their hemophilia A drug, those with the rare disease will eventually be treated effectively.

Hemophilia A is a hereditary bleeding disorder caused by a lack of blood clotting factor VIII. This causes the patient to experience extended bleeding that can cause pain and joint damage. The disease is caused by an inherited X-linked recessive trait and the defective gene is located on the X chromosome, resulting in a lack of factor VIII. Most people with hemophilia A are males because they only have one X-chromosome.

"The approval of this product provides an additional therapeutic option for use in the care of patients with hemophilia A," said Dr. Karen Midthun, director of the FDA's Center for Biologics Evaluation and Research, in this Boston Globe article. The drug will only require patients to inject themselves once every four or more days, compared to current medication that calls for injections once every two or three days.

The drug aims to treat patients who already use existing drugs to prevent their excessive bleeding as well as those who only treat symptoms and don't take medication or undergo treatment regularly. Eloctate is expected to help the former apply their injections less frequently with the same effect and encourage the latter to use the drug and get in a habit rather than using medications "on demand."

Additionally, the global market for hemophilia types A and B is estimated to be $7 billion per year, which was an obvious factor in Biogen's manufacturing of the drug. As of 2012, over 274,000 people across the world suffer from a form of hemophilia or other bleeding disorders, such as von Willebrand disease.