The U.S. Food and Drug Administration approved the first drug in almost 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 African American, 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.
Endari is made by a California company, Emmaus Medical Inc., and approved for adults and children 5 and older.
This treatment is one of only two drugs approved for the rare blood disorder.
Endari is taken as an oral powder.