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Treatment for Rare Bone Marrow Disease Gains FDA Approval

Jakafi bottles (Incyte Corp.)

(Incyte Corp.)

The Food and Drug Administration today approved the drug ruxolitinib, to treat the bone marrow disease myelofibrosis, for marketing in the U.S. Ruxolitinib is marketed under the brand name Jakafi by Incyte Corporation, a biotechnology company in Wilmington, Delaware.

Jakafi is a pill taken two times a day to inhibit enzymes called Janus Associated Kinase (JAK) 1 and 2 involved in regulating blood and immunological functioning. The disease myelofibrosis is associated with the deregulation of JAK 1 and 2, in which bone marrow is replaced by scar tissue resulting in blood cells being made in organs such as the liver and the spleen.

The disease is characterized by an enlarged spleen, anemia, and decreased white blood cells and platelets. Symptoms include fatigue, abdominal discomfort, pain under the ribs, feeling full (satiety), muscle and bone pain, itching, and night sweats.

FDA approval was based on results from two randomized phase 3 trials (COMFORT-I and COMFORT-II) that showed patients treated with Jakafi experienced significant reductions in splenomegaly (enlarged spleen). The COMFORT-I trial, conducted by Incyte, compared Jakafi to placebo in 309 patients with primary myelofibrosis, or myelofibrosis symptoms resulting from related disorders. The COMFORT-II trial, conducted by Novartis, Incyte’s partner outside of the U.S., compared Jakafi to best available therapy — either hydroxyurea, a chemotherapy agent, or glucocorticoids — in 219 patients.

A greater percentage of patients receiving Jakafi, says FDA, experienced more than a 35 percent reduction in spleen size when compared to patients receiving placebo or best available therapy. A greater proportion of patients receiving Jakafi also saw more than a 50 percent reduction in their myelofibrosis-related symptoms, including abdominal discomfort, night sweats, itching, and bone or muscle pain, than was the case in patients receiving placebo.

FDA says it reviewed Jakafi under the agency’s priority review program, an expedited six-month review of drugs that may offer significant advances in treatment over available therapy or that provide a treatment when no adequate therapy exists. Jakafi was also designated as an orphan drug, which treats a disease affecting fewer than 200,000 people in the U.S.

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