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News From the Food and Drug Administration
January 7, 2020

New Option for Sickle Cell Disease

JAMA. 2020;323(1):18. doi:10.1001/jama.2019.20640

The FDA has boosted treatment options for sickle cell disease by approving voxeletor for adults and adolescents aged 12 years or older who have the illness.

Marketed as Oxbryta, the drug “is an inhibitor of deoxygenated sickle hemoglobin polymerization, which is the central abnormality in sickle cell disease,” Richard Pazdur, MD, director of the FDA’s Oncology Center of Excellence, said in a statement. When patients take the medication “sickle cells are less likely to bind together and form the sickle shape, which can cause low hemoglobin levels due to red blood cell destruction,” Pazdur added.

Current treatments for sickle cell disease include the anticancer drug hydroxyurea, which increases fetal hemoglobin levels and decreases the frequency of painful episodes. In 2017, the FDA approved L-glutamine oral powder, an amino acid that helps prevent sickle red blood cells from sticking to endothelial cells in blood vessels. The only known cure is a bone marrow transplant, but it requires a matched donor and can cause severe adverse events. About 100 000 people in the United States have sickle cell disease, according to the FDA.