FDA approves drug for rare bone marrow disorder

SILVER SPRING, Md. — The Food and Drug Administration has approved a new drug for treating a rare bone marrow disease, the agency said Wednesday.

The FDA announced the approval of Incyte's Jakafi (ruxolitinib), calling it the first drug specifically approved for treating myelofibrosis, a disease in which the bone marrow is replaced by scar tissue, resulting in blood cells being made in the liver and spleen. The disease causes such symptoms as enlarged spleen, anemia and low white blood-cell count, as well as fatigue, muscle and bone pain, and pain and discomfort under the rips and in the abdominal area.

"Jakafi represents another example of an increasing trend in oncology where a detailed scientific understanding of the mechanisms of a disease allows a drug to be directed toward specific molecular pathways," FDA Office of Hematology and Oncology Products director Richard Pazdur said. "The clinical trials leading to this approval focused on problems that patients with myelofibrosis commonly encounter, including enlarged spleens and pain."

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