PNH Drugs and Treatments
More drugs and treatments are available to treat bone marrow failure disease than ever before. Researchers constantly look for ways to enhance drugs already on the market and develop new ones with fewer side effects. Some drugs treat only one type of the disease, while others may be used to treat more than one type. Check with your doctor to find out which drugs are right for you and why.
Pegcetacoplan
Brand name: EMPAVELI
EMPAVELI® is the first PNH treatment that binds to complement protein C3. It was approved by the Food and Drug Administration in May 2021 for treating adult patients with paroxysmal nocturnal hemoglobinuria (PNH). EMPAVELI is given skin (subcutaneously) by using the Empaveli injector or with an infusion pump 2 times a week.
Prednisone
Brand name: Rayos, Sterapred, Deltasone
Prednisone a man-made version of a hormone produced naturally by the adrenal gland that can help reduce inflammation and stop allergic responses. It is prescribed when the body is not producing enough of this chemical on its own.
Ravulizumab-cwvz
Brand name: Ultomiris
Ravulizumab-cwvz (Ultomiris®) is a drug approved by the U.S. Food and Drug Administration (FDA) in 2018 to treat PNH. ULTOMIRIS® is a long-acting C5 inhibitor that works by inhibiting the C5 protein in the terminal complement cascade. On June 7, 2021, the U.S. FDA approved the expanded use of ULTOMIRIS® to include children (one month of age and older) and adolescents with PNH.
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