Apr 26, 2023 · A Randomized, Multicenter, Active-Comparator Controlled, Open-Label Trial to Evaluate Efficacy and Safety of Oral, Twice Daily LNP023 in Adult Patients With PNH and Residual Anemia, Despite Treatment With an Intravenous Anti …

Aug 2, 2022 · Single-agent oral factor B inhibition led to rapid improvements of hemolytic markers, Hb levels, and transfusion requirements in PNH. Iptacopan (LNP023) is a novel, oral selective inhibitor of complement factor B under clinical development for paroxysmal nocturnal hemoglobinuria (PNH).

Basel, August 29, 2020 — Novartis today announced new Phase II data for LNP023, an investigational oral treatment for paroxysmal nocturnal hemoglobinuria (PNH), presented at the virtually held 2020 European Society for Blood and …

Basel, December 16, 2020 — Novartis today announced that the U.S. Food and Drug Administration (FDA) granted iptacopan (LNP023) Breakthrough Therapy Designation (BTD) in paroxysmal nocturnal hemoglobinuria (PNH) and Rare Pediatric Disease (RPD) Designation in C3 glomerulopathy (C3G).

Nov 5, 2021 · Iptacopan (LNP023) is a new, oral, selective and potent first-in-class inhibitor of factor B, a key component of the AP. Recent phase 2 data showed that iptacopan effectively controls both IVH and EVH and leads to rapid, transfusion-free improvements in Hb levels in the majority of PNH patients.

Mar 31, 2025 · The purpose of this open-label, single arm, multicenter, phase 3 study is to assess the pharmacokinetics of iptacopan in pediatric patients and to assess whether iptacopan is safe and well tolerated when used for the treatment of pediatric paroxysmal nocturnal hemoglobinuria (PNH) patients 2 to < 18 years of age.

6 days ago · Fabhalta is the first oral monotherapy for previously treated and treatment- naïve adults with paroxysmal nocturnal hemoglobinuria (PNH),1,2 a chronic, rare and potentially life-threatening blood disorder 3,4 Now available in Canada, this new treatment option helps address significant unmet needs for adult patients with PNH; a large proportion of patients can remain anemic and dependent on ...

LNP023 is a trial drug designed to block a protein in the complement system. Researchers think it may prevent or reduce hemolysis caused by PNH. • How many participants had a sign of hemolysis go down after receiving LNP023 for 12 weeks? • What medical problems did the participants have during this trial?

Iptacopan (LNP023) is a novel, oral selective inhibitor of complement factor B under clinical development for paroxysmal nocturnal hemoglobinuria (PNH). In this ongoing open-label phase 2 study, PNH patients with active hemolysis were randomized to ...

APPOINT-PNH study (NCT04820530) will assess iptacopan effi cacy and safety in PNH adult patients naïve to complement inhibitors, including anti-C5 MAbs. Design: The study comprises 8-wk screening; 24-wk, single-arm, open-label core treatment; and …