FDA approves first drug for bone marrow disorder

By MATTHEW PERRONE , AP Health Writer

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 twice-a-day pill Jakafi from Incyte Corp. was approved to treat , which causes anemia, fatigue, pain and swelling of the spleen. The disease spurs abnormal blood cells to build up in bone marrow, forming thick that slows the production of healthy blood cells. To make up for the shortage, other 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 , though some patients are not eligible for the procedure.

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, known as ruxolitinib, 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.

Jakafi is the first drug to reach the market from Wilmington, Del.-based Incyte. The company has partnered with Swiss drugmaker Novartis, which holds foreign marketing rights to the drug.

Incyte plans to launch Jakafi next week through specialty pharmacies throughout the U.S., according to a company statement.

Company shares rose $1.29, or 10.2 percent, to $13.89 in midday trading.

not rated yet
add to favorites email to friend print save as pdf

Related Stories

FDA adds heart warning to Pfizer anti-smoking pill

Jun 16, 2011

(AP) -- Federal health regulators are warning doctors and patients that Pfizer's anti-smoking drug Chantix may slightly increase the risk of heart attack and other cardiovascular problems.

Recommended for you

Powdered measles vaccine found safe in early clinical trials

8 hours ago

A measles vaccine made of fine dry powder and delivered with a puff of air triggered no adverse side effects in early human testing and it is likely effective, according to a paper to be published November 28 in the journal ...

Health care M&A leads global deal surge

Nov 23, 2014

In a big year for deal making, the health care industry is a standout. Large drugmakers are buying and selling businesses to control costs and deploy surplus cash. A rising stock market, tax strategies and ...

User comments

Please sign in to add a comment. Registration is free, and takes less than a minute. Read more

Click here to reset your password.
Sign in to get notified via email when new comments are made.