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Danicopan, sold under the brand name Voydeya, is a medication used for the treatment of paroxysmal nocturnal hemoglobinuria. It is a complement inhibitor which reversibly binds to factor D to prevent alternative pathway-mediated hemolysis and deposition of complement C3 proteins on red blood cells. The most common side effects include fever, headache, increased levels of liver enzymes (a sign ...
Hemoglobinuria; Other names: Haemoglobinuria: Structure of hemoglobin: Specialty: Urology, nephrology: Symptoms: Disease, Hemolytic anemia, Proteinuria: Causes: Acute glomerulonephritis; Burns; Renal cancer; Malaria; Paroxysmal nocturnal hemoglobinuria; Microangiopathies, e.g. hemolytic-uremic syndrome (HUS), thrombotic thrombocytopenic purpura (TTP) leading to microangiopathic hemolytic ...
Pegcetacoplan is the first treatment for paroxysmal nocturnal hemoglobinuria that binds to and inhibits complement protein C3. [9] Pegcetacoplan was approved for medical use in the United States in May 2021. [9] The US Food and Drug Administration (FDA) considers it to be a first-in-class medication. [16]
Other names: Thrombocytopaenia, thrombopenia ... Paroxysmal nocturnal hemoglobinuria; ... Other "blood thinners" sometimes used in this setting include bivalirudin ...
The drug crovalimab, branded as PiaSky, is a monthly under-the-skin or intravenous treatment for paroxysmal nocturnal hemoglobinuria (PNH). PNH is a disorder in which red blood cells break apart ...
Ravulizumab, sold under the brand name Ultomiris, is a humanized monoclonal antibody complement inhibitor medication designed for the treatment of paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome. It is designed to bind to and prevent the activation of Complement component 5 (C5). [8] [7]
Three phase III clinical trials have evaluated crovalimab in both people who were C5 inhibitor–naive and those switching to crovalimab from other C5 inhibitors. [10] COMMODORE 1 [11] is a phase III randomized clinical trial comparing crovalimab vs eculizumab in people with paroxysmal nocturnal hemoglobinuria treated with C5 inhibitors. [12]
Iptacopan, sold under the brand name Fabhalta, is a medication used for the treatment of paroxysmal nocturnal hemoglobinuria (PNH). [4] It is a complement factor B inhibitor that was developed by Novartis. [4] It is taken by mouth. [4]