The content discusses the approval of danicopan, a novel oral complement inhibitor, by the European Commission for the treatment of paroxysmal nocturnal hemoglobinuria (PNH). PNH is a rare, chronic, and progressive disease caused by a genetic mutation that leads to the expansion of abnormal blood cells deficient in glycosylphosphatidylinositol-linked proteins. This results in complement activation, intravascular hemolysis, and various complications such as thrombosis, infections, and bone marrow failure.
The standard treatment for PNH is complement blockade using anti-C5 monoclonal antibodies like ravulizumab or eculizumab. However, 10-20% of treated patients still experience clinically significant extravascular hemolysis and residual hemolytic anemia. Danicopan, a selective inhibitor of complement factor D, has been approved as an add-on therapy to the standard complement blockade to address this unmet need.
The approval is based on the results of the ongoing phase 3 ALPHA trial, which showed that the addition of danicopan to ravulizumab or eculizumab resulted in a statistically significant increase in mean hemoglobin levels at 12 weeks, along with reduced fatigue, anemia, and transfusion requirements. Danicopan was generally well-tolerated, with no serious adverse events reported.
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by Dr Sheena Me... at www.medscape.com 04-23-2024
https://www.medscape.com/viewarticle/europe-approves-paroxysmal-nocturnal-hemoglobinuria-drug-2024a10007ttDeeper Inquiries