FDA approves first drug for bone marrow disorder

November 16, 2011 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.

Explore further: To treat rare disease, NIH scientists repurpose FDA-approved drug

shares

Related Stories

To treat rare disease, NIH scientists repurpose FDA-approved drug

September 2, 2011
A new study reports that a drug already approved by the Food and Drug Administration for use in patients undergoing a bone marrow transplant may also have promise for treating people who have a rare immune deficiency known ...

Recommended for you

Study suggests ending opioid epidemic will take years

July 20, 2017
The question of how to stem the nation's opioid epidemic now has a major detailed response. A new study chaired by University of Virginia School of Law Professor Richard Bonnie provides extensive recommendations for curbing ...

Team-based model reduces prescription opioid use among patients with chronic pain by 40 percent

July 17, 2017
A new, team-based, primary care model is decreasing prescription opioid use among patients with chronic pain by 40 percent, according to a new study out of Boston Medical Center's Grayken Center for Addiction Medicine, which ...

Private clinics' peddling of unproven stem cell treatments is unsafe and unethical

July 7, 2017
Stem cell science is an area of medical research that continues to offer great promise. But as this week's paper in Science Translational Medicine highlights, a growing number of clinics around the globe, including in Australia, ...

Popular heartburn drugs linked to higher death risk

July 4, 2017
Popular heartburn drugs called proton pump inhibitors (PPIs) have been linked to a variety of health problems, including serious kidney damage, bone fractures and dementia. Now, a new study from Washington University School ...

Most reproductive-age women using opioids also use another substance

June 30, 2017
The majority of reproductive-age and pregnant women who use opioids for non-medical purposes also use at least one other substance, ranging from nicotine or alcohol to cocaine, according to a University of Pittsburgh Graduate ...

At-risk chronic pain patients taper opioids successfully with psychological tools

June 28, 2017
Psychological support and new coping skills are helping patients at high risk of developing chronic pain and long-term, high-dose opioid use taper their opioids and rebuild their lives with activities that are meaningful ...

0 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.