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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Thank you for keeping us inform, how can we get for the Dominican Republic?
Warm Regards,
Angela Mercado
MPH 2020
Monroe College
How can we get it in Nigeria
I have a sickle child 9 years she is now on hydroxcurea 500mg daily. How long will take the medicine? Is there anything to cure the disease? For now i am giving her dates and milk juice daily and her Haemoglobin is 11.5
If it really does make oxygen bind more tightly to hemoglobin, it sounds like just what is needed (in combination with inhaled oxygen) for immediate treatment of carbon monoxide poisoning in the general population. Something like an epi-pen, but with this drug instead.
I think that is very unlikely that a solid oral dose would be used for that acute situation. I have not read any information about this drugs PK profile but I think it much more likely it works over the course of weeks.