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Novartis Presents P-III Trial (APPOINT-PNH) Results of Iptacopan for the Treatment of Paroxysmal Nocturnal Hemoglobinuria at EBMT 2023

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Novartis Presents P-III Trial (APPOINT-PNH) Results of Iptacopan for the Treatment of Paroxysmal Nocturnal Hemoglobinuria at EBMT 2023

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  • The P-III trial (APPOINT-PNH) evaluating iptacopan (200mg, BID) in 40 adult patients. The trial met its 1EPs & showed a clinical benefit across 2EPs where 92.2% & 62.8% achieved a ≥2g/dL & ≥12 g/dL Hg-level increase from baseline without the need for RBC transfusions after 24wk.
  • 97.6% achieved RBC transfusion independence @24wks. with no clinical breakthrough hemolysis events or MAVEs, 83.55% reduction of LDH levels with the reduction seen as early as 7 Days, 95% had LDH levels ≤1.5 times the ULN along with improved patient-reported fatigue
  • The results were consistent with the tolerability & safety profiles as seen in the (APPLY-PNH) trial. The P-III trial (APPLY-PNH) results were also presented which showed the superiority of iptacopan over anti-C5 therapy

Ref: Novartis Image: Novartis

Related News:- Novartis Presents P-III Trial (APPLY-PNH) Result of Iptacopan for Paroxysmal Nocturnal Hemoglobinuria at ASH 2022

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Neha is a Senior Editor at PharmaShots. She is passionate and very enthusiastic about recent updates and developments in the life sciences and pharma industry. She covers Biopharma, MedTech, and Digital health segments along with different reports at PharmaShots. She can be contacted at connect@pharmashots.com.

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