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.
Deferiprone
Brand name: Ferriprox
Deferiprone is in a class of medications called iron chelators. It works by attaching to iron in the body so that it can be excreted (removed from the body).
Deferoxamine
Brand name: Desferal
Deferoxamine is an iron-binding agent that belongs to a class of drugs known as heavy metal antagonists. It works by helping the kidneys and gallbladder get rid of the extra iron.
Eculizumab
Brand name: Soliris
Eculizumab (Soliris®) was the first drug approved by the U.S. Food and Drug Administration (FDA) and the European Medicines Evaluation Agency (EMEA) to treat PNH. It works by making your complement system less active and reduces hemolysis Soliris® is approved for the treatment of patients with PNH in nearly 50 countries worldwide.
Enoxaparin
Brand name: Lovenox
Enoxaparin is in a class of medications called low molecular weight heparins. It works by stopping the formation of substances that cause clots. It is also used in combination with warfarin to treat blood clots in the leg.
Filgrastim
Brand name: G-CSF, Neupogen
A man-made version of a naturally occurring substance in the body that promotes white blood cell growth in the bone marrow.
Fludarabine
Brand name: Fludara
An antineoplastic agent, this medicine works by slowing or stopping the growth of cancer cells.
Folate and Folic Acid
Folate is a B-vitamin that is found in fresh or lightly cooked green vegetables. It helps your bone marrow make normal blood cells. When your bone marrow has to make more cells, it needs a larger supply of folic acid. Most people get enough folate in their diet. But if you have PNH, it's a good idea to take 1 mg each day of a man-made form of folate called folic acid.
Heparin
Brand name: Calciparine, Liquaemin
Heparin is in a class of medications called anticoagulants ('blood thinners'). It works by decreasing the clotting ability of the blood. Heparin is also used to stop the growth of clots that have already formed in the blood vessels, but it cannot be used to decrease the size of clots that have already formed.
Iptacopan
Brand name: Fabhalta
FABHALTA, a complement factor B inhibitor, is the first oral medication approved to treat adults with paroxysmal nocturnal hemoglobinuria (PNH). It was approved by the U.S. Food and Drug Administration in December 2023. Fabhalta is taken twice a day in a capsule form.
Iron Chelation
Iron chelation therapy is the main treatment used when you have a condition called iron overload. Iron overload means you have too much iron in your body. This can be a problem for people who get lots of red blood cell transfusions.