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. 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. 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.