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Iptacopan Meets Primary End Point of APPOINT-PNH Trial in Paroxysmal Nocturnal Hemoglobinuria

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. ....

France General , David Soergel , Antonio Risitano , Giuseppe Moscati , Reference Center , Greater Paris University Hospital , Interest Group , Metabolism Development Unit , Functional Assessment , Chronic Illness Therapy , Hematopoietic Transplant Unit , Aplastic Anemia , Paroxysmal Nocturnal Hemoglobinuria , Peffault De Latour , Saint Louis Hospital , Complement Inhibitor Naïve Paroxysmal Nocturnal Hemoglobinuria ,

Trial Shows Iptacopan Lowers Need for Blood Transfusion in Almost All Patients With Paroxysmal Nocturnal Hemoglobinuria

The APPOINT-PNH study showed that twice-daily LNP023 (Iptacopan) helped patients with paroxysmal nocturnal hemoglobinuria control intravascular hemolysis. ....

David Soergel , Giuseppe Moscati , Antonio Risitano , Metabolism Development Unit , Interest Group , Meeting Of The European Society For Blood , Reference Center , Annual Meeting , European Society , Marrow Transplantation , Hematopoietic Transplant Unit , Aplastic Anemia , Paroxysmal Nocturnal Hemoglobinuria , Functional Assessment , Chronic Illness Therapy ,

Novartis Phase III APPOINT-PNH trial shows investigational

Data at EBMT show primary endpoint met – estimated 92.2% of complement-inhibitor-naïve patients with paroxysmal nocturnal hemoglobinuria (PNH) achieving 2. ....

South Korea , United States , France General , Sloan Simpson , Parag Mahanti , Ann Hematol , Richard Jarvis , Iptacopan Monotherapy , Isabella Zinck , Samir Shah , Nicole Zinsli Somm , Mary Carmichael , David Soergel , Satoshi Sugimoto , Julie Masow , Michael Meo , Giuseppe Moscati , Antonio Risitano , Marlena Abdinoor , Value Health , Greater Paris University Hospital , Interest Group , Novartis Institutes For Biomedical Research , Metabolism Development Unit , Exchange Commission , Reference Center ,

Novartis Pharma AG: Novartis Phase III APPOINT-PNH trial shows investigational oral monotherapy iptacopan improves hemoglobin to near-normal levels, leading to transfusion independence in all treatment-naïve PNH patients

Data at EBMT show primary endpoint met - estimated 92.2% of complement-inhibitor-naïve patients with paroxysmal nocturnal hemoglobinuria (PNH) achieving 2 g/dL or more hemoglobin-level increase from baseline ....

South Korea , France General , United States , Satoshi Sugimoto , Julie Masow , Giuseppe Moscati , Samir Shah , David Soergel , Ann Hematol , Mary Carmichael , Antonio Risitano , Iptacopan Monotherapy , Marlena Abdinoor , Michael Meo , Nicole Zinsli Somm , Isabella Zinck , Richard Jarvis , Sloan Simpson , Parag Mahanti , Value Health , Metabolism Development Unit , Greater Paris University Hospital , European Medicines Agency , Novartis Institutes For Biomedical Research , Interest Group , Novartis Pharmaceuticals ,

Trial data released for potentially "practice-changing" PNH medicine

Iptacopan achieved "improvement of anaemia never seen before with anti-C5s” in PNH, showed Novartis data presented at 2023 EBMT. ....

France General , David Soergel , Antonio Risitano , Giuseppe Moscati , Interest Group , Meeting Of The European Society For Blood , Reference Center , Metabolism Development Unit At Novartis , Greater Paris University Hospital , European Society , Marrow Transplantation , Peffault De Latour , Saint Louis Hospital , Greater Paris University , Hematopoietic Transplant Unit , Aplastic Anemia , Paroxysmal Nocturnal Hemoglobinuria , Global Head , Metabolism Development Unit ,