First New Drug in 20 Years for Sickle Cell Approved
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The U.S. Food and Drug Administration has approved the first drug in nearly 20 years for sickle cell, an inherited disease in which abnormally shaped red blood cells can’t properly carry oxygen throughout the body, which can cause severe pain and organ damage.
About 100,000 people in the U.S., mostly blacks, have the disorder and about 275,000 babies are born with it each year worldwide.
In a study, the new drug, Endari, cut the number of pain crises and dangerous chest complications, and reduced hospitalizations and the need for transfusions.
It is made by a California company, Emmaus Medical Inc., and approved for adults and children 5 and older.