US approves drug to prevent sickle cell pain, organ damage

U.S. regulators have approved a new medicine that can prevent some extremely painful sickle cell disease flare-ups.

The Food and Drug Administration on Friday approved Novartis’s Adakveo (ah-DAHK’-vee-oh) for patients 16 and older. The monthly infusion halves occurrences of pain episodes.

Sickle cell disease is one of the most common inherited blood disorders and affects about 100,000 Americans, mostly blacks.

The pain episodes happen when misshapen blood cells clump together, blocking blood and oxygen flow. The flare-ups steadily damage organs and often require hospitalization. They also can kill suddenly, though most patients now live into their 40s.

The Novartis drug has a list price between $85,000 and $113,000 a year, based on dosing. Insured patients generally will pay less.

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