FDA okays Jakafi, first drug for rare marrow disease

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(CBS/AP) The first drug for a rare bone marrow disease was approved Wednesday by the FDA. Jakafi (ruxolitinib) is a twice-a-day pill for myelofibrosis, in which a buildup of abnormal red blood cells inside marrow causes anemia, fatigue, pain, night sweats, and swelling of the spleen.

The drug's maker, Delaware-based Incyte, estimates that myelofibrosis affects 16,000 to 18,500 people in the U.S., though precise figures are unavailable. Currently, the disease is treated with chemotherapy or bone marrow transplantation, and some patients are ineligible for the procedures.

Incyte said Wednesday that Jakafi will cost $7,000 a month, or $84,000 for a year's supply for insured patients. The company plans to provide the drug free to uninsured patients and will offer copay assistance to patients with financial need.

The FDA approved the drug based on two studies that included 528 patients with the disease. Patients were randomly assigned to receive a placebo or Jakafi. More patients in the drug group saw a significant reduction in the size of their spleen as well as a 50 percent decrease in symptoms.

The FDA reviewed Jakafi under its priority review program for important new therapies, which aims to clear drugs in six months instead of the usual 10.

Jakafi works by blocking two enzymes associated with the myelofibrosis. Side effects reported in patients taking the drug included diarrhea, headache, dizziness and nausea.

The Mayo Clinic has more on myelofibrosis.

  • David W Freeman

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