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New Treatment to Prevent Sickle Cell Crisis

New Treatment to Prevent Sickle Cell Crisis

New Treatment to Prevent Sickle Cell Crisis

On November 15, 2020, the U.S. Food and Drug Administration approved Adakveo®, a treatment to reduce the frequency of vaso-occlusive crisis (VOC) in patients with sickle cell disease. These severe events are unpredictable and can cause life-threatening complications.

Sickle cell disease is an inherited blood disorder in which the red blood cells are abnormally shaped (in a crescent or “sickle” shape), which restricts the flow in blood vessels and limits oxygen delivery to the body’s tissues, leading to severe pain and organ damage. VOC is an extremely painful complication of sickle cell disease that occurs when blood circulation is obstructed by sickled red blood cells. Sickle cell disease affects approximately 100,000 Americans. The disease occurs most often in African-Americans, affecting 1 out of every 365 babies.

Adakveo® works by inhibiting selectin, a substance that contributes to cells sticking together and leads to vaso-occlusive crisis. In clinical trials, Adakveo® reduced the median annual rate of VOCs leading to health care visits by 45.3% compared with placebo.

Adakveo® is an IV infusion administered to patients once per month and has a wholesale acquisition cost of $2,357 per vial. Most patients will require between 3 and 4 vials per month. This would make the annual list price between $84,000 and $115,000.

Additional Information: FDA, Novartis, FiercePharma