Recently, the U.S. Food and Drug Administration approved Adakveo and Oxbryta, two new drugs used in the treatment of sickle cell disease for both adult and pediatric patients.

Published Dec 19th, 2019

By Nichelle Smith

nsmith@health.southalabama.edu

Recently, the U.S. Food and Drug Administration approved Adakveo and Oxbryta, two new drugs used in the treatment of sickle cell disease for both adult and pediatric patients.

FDA approval for the two drugs was based on the results of two separate randomized clinical trials. Johnson Haynes, M.D., director of the USA Comprehensive Sickle Cell Center, said the center played an active role in the evaluation phase of approval enrolling patients in Phase III clinical trials for both drugs.

“These are exciting times with the FDA recent approval of these new medications. We now have a total of four drugs that can positively impact the care of those living with sickle cell disease,” he said.

Sickle cell disease is an inherited blood disorder in which the red blood cells are abnormally shaped, restricting the flow in blood vessels and limiting oxygen delivery to the body’s tissues.

The first drug, Adakveo, works to treat vaso-occlusive crises – a common and painful complication of sickle cell disease that occurs when blood circulation is obstructed by sickled red blood cells. “Adakveo is the first non-opioid with a clinical indication of pain management in patients living with sickle cell disease,” Haynes said.

According to Haynes, Oxbryta - which was approved for the treatment of sickle cell disease in adults and pediatric patients 12 years of age or older- is the first drug to specifically address the root cause of the disease. The FDA reports that nonclinical studies have demonstrated that Oxbryta inhibits red blood cell sickling, improves red blood cell deformability and improves the blood’s ability to flow.

Acting FDA Commissioner Adm. Brett P. Grior, M.D., said the approval provides additional hope to the 100,000 people in the United States, and the more than 20 million globally who live with this debilitating blood disorder. “Our scientific investments have brought us to a point where we have many more tools available in the battle against sickle cell disease, which present daily challenges for those living with it,” he said. “We remain committed to raising the profile of this disease as a public health priority and to approving new therapies that are proven to be safe and effective. Together with improved provider education, patient empowerment, and improved care delivery systems, these newly approved drugs have the potential to immediately impact people living with sickle cell disease.”

Learn more about Oxbryta and Adakveo.

To learn more about USA Health Comprehensive Sickle Cell Center, click here

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