Iptacopan Meets Primary End Point of APPOINT-PNH Trial in Pa

Iptacopan elicited improved hemoglobin levels measuring at least 2 g/dL higher vs baseline, leading to transfusion independence after 24 weeks in approximately 92.2% of patients with complement inhibitor–naïve paroxysmal nocturnal hemoglobinuria.

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Italy , Paris , France General , France , Antonio Risitano , Giuseppe Moscati , David Soergel , Metabolism Development Unit , Novartis , Interest Group , Reference Center , Greater Paris University Hospital , Functional Assessment , Chronic Illness Therapy , Hematopoietic Transplant Unit , Aplastic Anemia , Paroxysmal Nocturnal Hemoglobinuria , Peffault De Latour , Saint Louis Hospital , Iptacopan , Pnh , Appoint Pnh Trial , Nct04820530 ,