The Food and Drug Administration on Monday approved a once-daily pill for sickle cell disease that works in an entirely new way — by boosting hemoglobin, the oxygen-carrying molecule found in red blood cells.
The novel drug was developed by the biotech firm Global Blood Therapeutics and will be sold under the brand name Oxbryta. The drug’s approval came three months earlier than expected.
Sickle cell disease is an inherited disorder that affects about 100,000 people in the U.S., mainly African Americans. The disease results from a mutation in the gene for hemoglobin. Oxbryta works by making oxygen attach more tightly to hemoglobin. By keeping hemoglobin in a highly oxygenated state, the mutation that causes sickle cell disease is covered up, helping the red blood cells maintain a normal shape and preventing them from clumping together inside blood vessels.
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