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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 ...
The sucrose lysis test is a diagnostic laboratory test used for diagnosing paroxysmal nocturnal hemoglobinuria (PNH), as well as for hypoplastic anemias and any hemolytic anemia with an unclear cause. [1] The test works by using sucrose, which creates a low ionic strength environment that allows complement to bind to red blood cells. [1]
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]
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]
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]
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 ...
COMMODORE 3 [19] is a phase III single-arm trial run in China, studying crovalimab in C5 inhibitor-naive people with paroxysmal nocturnal hemoglobinuria. [20] COMMODORE 3 assessed the safety, efficacy, pharmacokinetics, and pharmacodynamics of crovalimab in people with C5-naive paroxysmal nocturnal hemoglobinuria.
It was initially approved to treat paroxysmal nocturnal hemoglobinuria, a rare blood disorder. [7] In June 2010, there was an outbreak of hemolytic–uremic syndrome caused by shigatoxigenic and verotoxigenic Escherichia coli (EHEC) in Germany.