FDA
TALK PAPER

Food and Drug Administration
U.S. Department of Health and Human Services
Public Health Service 5600 Fishers Lane Rockville, MD 20857
FDA Talk Papers are prepared by the Press Office to guide FDA personnel in responding with consistency and accuracy to questions from the public on subjects of current interest. Talk Papers are subject to change as more information becomes available. 
T98-11                        Print Media:        301-827-6242
March 4, l998                 
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FDA APPROVES DROXIA FOR SICKLE CELL ANEMIA

FDA has approved a new indication for the decades old cancer drug hydroxyurea (Droxia) -- as a treatment for adults who suffer from sickle cell anemia. Sickle cell anemia is a rare, inherited blood disorder that causes chronic anemia and periodic episodes of pain. About 91,000 people in the United States, mostly of African-American heritage, are affected by the disease. The following may be used to respond to questions.

Droxia has been approved -- specifically for patients over 18 who have had at least three "painful crises" in the previous year -- to reduce the frequency of these crises and the need for blood transfusions. Sickle cell anemia causes red blood cells to become brittle and sickle shaped. During a painful crisis, the sickle shaped red blood cells get stuck in narrow blood vessels, blocking the flow of blood and oxygen to the body's tissues. These painful crises can cause pain and swelling of hands and feet, along with fever, fatigue, shortness of breath, eye problems, acute chest syndrome (a pneumonia-like condition that leads to difficulty breathing), and infections.

Although the drug is not a cure for sickle cell disease, it may help control the symptoms of the disease. Doctors establish the drug dose based on the patient's weight and blood count. While taking the drug, patients must have a blood test every two weeks to ensure that their blood count is not depressed to a level at which they might be at risk for infection or bleeding. Almost all patients who received Droxia in the clinical trials needed to have their medication stopped for a time to allow their low blood count to return to acceptable levels.

Because sickle cell anemia affects a small population of patients, Droxia has been designated as an orphan drug. Orphan drug status guarantees the developer of an orphan product seven years of market exclusivity for the orphan drug indication following the marketing approval by the FDA, and provides other financial incentives for drug development.

Droxia (hydroxyurea) is manufactured by Bristol Laboratories, Princeton, NJ. (Hydroxyurea is also approved by FDA under the name Hydrea for treatment of certain types of leukemia and other cancers.)

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