Long-term efficacy and safety of danicopan as add-on therapy to ravulizumab or eculizumab in PNH with significant EVH
In this medfyle
The phase 3 ALPHA trial highlights the potential of combining danicopan with ravulizumab or eculizumab for PNH. Long-term data over 72 weeks confirmed sustained efficacy, with improved fatigue scores and no new safety signals. This combination treatment offers effective control of terminal complement activity, intravascular hemolysis, and clinically significant extravascular hemolysis, providing new hope for those with persistent anemia in PNH.
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This Medfyle is provided as a service to healthcare professionals. It has been created from content originally published in Blood, American Society of Hematology's official journal.
Original article: Kulasekararaj A, Griffin M, Piatek C, Shammo J, Nishimura JI, Patriquin C, Schrezenmeier H, Barcellini W, Panse J, Gaya A, Patel Y, Liu P, Filippov G, Sicre de Fontbrune F, Risitano A, Lee JW. Long-term efficacy and safety of danicopan as add-on therapy to ravulizumab or eculizumab in PNH with significant EVH. Blood. 2025 Feb 20;145(8):811-822.
