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.
#FDAapproves new treatment for sickle cell disease, first approval for this rare blood disorder in nearly 20 years https://t.co/15cdb8qt9x
— U.S. FDA (@US_FDA) 7 de julio de 2017
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 five and older.