FDA approves first drug for bone marrow disorder

WASHINGTON (AP) ? The first drug to treat a rare disorder that causes red blood cells to build up inside bone marrow was cleared Wednesday by the Food and Drug Administration.

The FDA approved the twice-a-day pill Jakafi from Incyte Corp. to treat myelofibrosis, which causes anemia, fatigue, pain and swelling of the spleen. The disease spurs abnormal blood cells to build up in bone marrow, forming thick scar tissue that slows the production of healthy blood cells. To make up for the shortage, other organs including the liver and the spleen begin producing blood cells.

Incyte estimates the disease affects between 16,000 and 18,500 people in the U.S., though precise figures are not available. The disease is currently treated with chemotherapy or bone marrow transplant, and some patients are not eligible for the procedures.

Incyte said it plans to launch Jakafi nationwide next week through specialty pharmacies.

Known generically as ruxolitinib, Jakafi is the first drug to reach the market from Wilmington, Del.-based Incyte Corporation. The company has partnered with Swiss drugmaker Novartis, which holds the rights to market Jakafi abroad. The company said Wednesday that Jakafi will cost $7,000 per month, or $84,000 for a year's supply for insured patients. Incyte plans to provide the drug free to uninsured patients and will offer co-pay assistance to patients with financial need.

The FDA approved the drug based on two studies including 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, including pain, discomfort and night sweats.

The drug works by blocking two enzymes associated with the disease.

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.

Side effects reported in patients taking the drug included diarrhea, headache, dizziness and nausea.

Company shares rose 97 cents, or 7.7 percent, to $13.57 in midday trading.

Associated Press

Source: http://hosted2.ap.org/APDEFAULT/bbd825583c8542898e6fa7d440b9febc/Article_2011-11-16-Incyte%20Drug-FDA%20Approval/id-e0767f1d984a450ab337847520b21752

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