Colorized microscope image showing cells (© Janice Haney Carr/CDC/Sickle Cell Foundation of Georgia)
A new treatment makes cells less likely to form the sickle shape seen on the left. (© Janice Haney Carr/CDC/Sickle Cell Foundation of Georgia)

U.S. regulators have approved a California company’s new therapy for treating sickle cell disease, a painful and life-threatening inherited blood disorder.

“Today’s approval provides additional hope to the 100,000 people in the U.S., and the more than 20 million globally, who live with this debilitating blood disorder,” Brett P. Giroir, acting commissioner of the U.S. Food and Drug Administration, said November 25, when the agency approved Oxbryta for patients 12 and older.

Global Blood Therapeutics Inc., a San Francisco-based biopharmaceutical company, says its Oxbryta is the first approved drug that targets the blood cell malformation that is the root cause of the disease.

In patients with sickle cell disease, red blood cells change to the shape of a sickle, hampering blood flow. The disease primarily affects people in or descended from sub-Saharan Africa.

Richard Pazdur, a senior FDA researcher, says, “With Oxbryta, 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.”

The FDA granted “accelerated approval” of Oxbryta, which means the drug can be used to fill an unmet medical need but that additional clinical trials are still required.

GBT calls Oxbryta “a major milestone” for people living with sickle cell disease.