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Novartis Phase III APPOINT-PNH trial shows investigational - Vimarsana News

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...

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 - Vimarsana News

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

Novartis Pharma AG: Novartis presents pivotal Phase III APPLY-PNH data at ASH demonstrating investigational oral monotherapy iptacopan superiority over anti-C5 - Vimarsana News

Novartis Pharma AG: Novartis presents pivotal Phase III APPLY-PNH data at ASH demonstrating investigational oral monotherapy iptacopan superiority over anti-C5

Trial met both primary and most secondary endpoints, showing iptacopan provided transfusion-free hemoglobin-level increases in vast majority of adult paroxysmal nocturnal hemoglobinuria (PNH) patients

Novartis investigational iptacopan provides clinically meaningful increases in hemoglobin levels in complement-inhibitor-naïve patients with PNH - Vimarsana News

Novartis investigational iptacopan provides clinically meaningful increases in hemoglobin levels in complement-inhibitor-naïve patients with PNH

Phase III APPOINT-PNH study of investigational oral monotherapy iptacopan met its primary endpoint; second positive Phase III topline readout for iptacopan in...

Novartis investigational oral therapy iptacopan (LNP023) receives FDA Breakthrough Therapy Designation for PNH and Rare Pediatric Disease Designation for C3G - Vimarsana News

Novartis investigational oral therapy iptacopan (LNP023) receives FDA Breakthrough Therapy Designation for PNH and Rare Pediatric Disease Designation for C3G

Posted December 16th, 2020 for Novartis Paroxysmal nocturnal hemoglobinuria (PNH) is a rare and life-threatening blood disorder, resulting in debilitating symptoms that can impact patients’ quality of life 1–3 C3 glomerulopathy (C3G) is a rare renal disease, affecting young patients with a poor prognosis and significant unmet need 4-5 With potential to be the first oral treatment for a range of complement-driven diseases, complement factor B inhibitor iptacopan targets the underlying cause of these conditions through its action on the complement system’s alternative pathway 6,7 ...