Jakafi Gets FDA Approval For Chronic Bone Marrow Condition

The U.S. Food and Drug Administration (FDA) recently approved a new drug to treat patients with polycythemia vera, a chronic bone marrow condition. The new drug, Jakafi (ruxolitnib), is the first to be approved by the FDA for this health problem.

"The approval of Jakafi for polycythemia vera underscores the importance of developing drugs matched to our increasing knowledge of the mechanisms of diseases," said Richard Pazdur, M.D., director of the Office of Hematology and Oncology Products in the FDA's Center for Drug Evaluation and Research. "The trial used to evaluate Jakafi confirmed clinically meaningful reductions in spleen size and the need for phlebotomies to control the disease."

The condition occurs when an excessive number of red blood cells are created in the bone marrow-causing patients to suffer from an increase in white blood cell platelets. This can cause the spleen to swell with bleeding problems and blood clots if it is not properly treated.

However, some patients have been unable to tolerate hydroxyurea, which helps to lower the number of red blood cells and platelets in the blood. The new drug works by inhibiting enzymes called Janus Associated Kinase (JAK) 1 and 2 that help to regulate blood and immunological function. It can also help to decrease the occurrence of enlarged spleens.

For a clinical study, researchers examined 222 participants who had the disease for at least 24 weeks and did not respond well to hydroxyurea and had undergone a phlebotomy procedure and exhibited symptoms of an enlarged spleen. All participants were either assigned to receive Jakafi or another therapy treatment.

Findings revealed that about 21 percent of Jakafi-treated participants exhibited a reduction in the need for a phlebotomy. The most common side-effects were anemia (or low red blood cell counts) and low blood platelet counts (thrombocytopenia).