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FDA Approves Iptacopan (Fabhalta) for Paroxysmal Nocturnal Hemoglobinuria

The approval was based on data from the APPLY-PNH trial and APPOINT-PNH study, which showed iptacopan was superior for hemoglobin improvement in the absence of red blood cell transfusions and in transfusion avoidance rate compared to patients who stayed on anti-C5 treatments. ....

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