FDA approves second treatment for sickle cell disease

The FDA has approved Global Blood Therapeutics’ drug, Oxbryta, designed to treat sickle cell disease in patients 12 years and older. 

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The Nov. 25 approval came just 10 days after the FDA approved Novartis’ drug, Adakveo, the first targeted therapy to treat the disease for patients 16 years and older. 

Sickle cell disease is a lifelong inherited blood disorder in which red blood cells are abnormally shaped like a crescent, restricting blood flow and limiting oxygen delivery to the body’s tissues. The disease causes severe pain and organ damage. 

Oxbryta was shown in a clinical trial to inhibit red blood cell sickling and improve the blood’s ability to flow. 

Common side effects of Oxbryta were headache, diarrhea, abdominal pain, nausea, fatigue, rash and fever.

Oxbryta was granted accelerated approval, which is reserved for drugs treating serious conditions to fill an unmet medical need. It was also granted fast-track designation and orphan drug designation.

Read the full news release here

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