The factor D inhibitor danicopan has been approved for use in Europe for patients with paroxysmal nocturnal hemoglobinuria with residual hemolytic anemia on standard therapy.
The first-in-class complement factor D inhibitor danicopan has been approved by the Europe an Commission as an add-on agent for certain patients with the rare disease. The mutation results in the expansion of a clone of hematopoietic cells that are deficient in glycosylphosphatidylinositol-linked proteins.
Intravascular hemolysis leads to release of free hemoglobin into the blood and toxic effects including hypercoagulability, changes in vascular tone from reduction of circulating nitric oxide, and renal damage. Extravascular hemolysis also occurs because complement component 3 accumulates on the surface of the affected erythrocytes, leading to opsonization with reticuloendothelial destruction in the liver and spleen., dyspnea, fatigue, anemia, and dark-colored urine.
European Commission approval of danicopan as an addition to ravulizumab or eculizumab in adult patients with PNH and continuing clinically significant EVH follows a positive opinion of the Committee for Medicinal Products for Human Use in February and is based on the protocol-prespecified interim efficacy analysis of results published in December 2023 from the first 63 patients in the ongoing
ALPHA showed that addition of danicopan resulted in a statistically significant increase in mean hemoglobin level at 12 weeks . This was associated with reduced fatigue and anemia and a reduced need for transfusions. The drug was generally well tolerated, with no serious adverse events reported.All material on this website is protected by copyright, Copyright © 1994-2024 by WebMD LLC. This website also contains material copyrighted by 3rd parties.
PNH - Paroxysmal Nocturnal Hemoglobinuria Paroxysmal Nocturnal Haemoglobinuria Paroxysmal Nocturnal Hemoglobinuria (PNH) Europe European Hemolytic Anemia Haemolytic Anaemia Anemia Anaemia Hemolysis Biologic Therapy Biologics Genomics Genomic Medicine Orphan Drugs Rare Diseases
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