FDA Approves Treatment for Chronic Type of Bone Marrow Disease

The FDA today approved ruxolitinib (Jakafi) to treat patients with polycythemia vera, a chronic type of bone marrow disease.

Patients with polycythemia vera have too many red blood cells in their bone marrow, and they may also experience an increase in white blood cells and platelets. Patients’ spleens can swell as a consequence, and they may have bleeding problems and blood clots in the veins near the skin surface. Patients with polycythemia vera are also more prone to stroke or heart attack.

The only FDA-approved drug to treat the disease, Jakafi is intended to help patients who cannot tolerate, or have an inadequate response to, hydroxyurea, which is another medicine used to reduce the number of red blood cells and platelets in the blood, according to the FDA.

Jafaki inhibits enzymes called Janus Associated Kinase 1 and 2, which are involved in regulating blood and immunological functioning, according to the FDA. With Jafaki, there will be less of a need for phlebotomy, which removes excess blood from the body. It will also decrease the occurrence of an enlarged spleen, according to the FDA.

More than 200 patients participated in a clinical study, which showed 21% of Jakafi-treated participants saw a reduction in the need for a phlebotomy and a reduction in spleen volume, compared with 1% of participants who received the best available therapy.

“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, MD, director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research, in a press release. “The trial used to evaluate Jakafi confirmed clinically meaningful reductions in spleen size and the need for phlebotomies to control the disease.”