The FDA has approved danicopan as an add-on therapy for patients with paroxysmal nocturnal hemoglobinuria (PNH) who are receiving ravulizumab or eculizumab. PNH is a rare blood disorder that can result in life-threatening complications such as anemia, thrombosis, and bone marrow dysfunction. Danicopan, an oral complement factor D inhibitor, helps control intravascular hemolysis and prevent extravascular hemolysis. The approval was based on a phase 3 trial showing that danicopan significantly improved hemoglobin concentrations and reduced the need for blood transfusions. Common side effects include headache, nausea, and arthralgia. Danicopan carries a black box warning for serious infections and is only available through a Risk Evaluation and Mitigation Strategy program.
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FDA OKs Danicopan for Extravascular Hemolysis in PNH
