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).

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Iptacopan (LNP023) is a first-in-class, orally bioavailable, highly potent and highly selective factor B inhibitor with an IC50 value of 10 nM. Iptacopan shows direct, reversible, and high-affinity binding to human factor B with a KD of 7.9 nM.

2022年8月2日 · 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).

2021年11月5日 · 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.

2025年2月3日 · The study is designed as a multicenter, single-arm, open label study to demonstrate the efficacy and safety of LNP023 (iptacopan) at a dose of 200 mg b.i.d. in adult patients with aHUS who are treatment naive to complement …

Cohort A: To evaluate the efficacy of iptacopan (LNP023) in reducing proteinuria at Week 12 measured as ratio to baseline of UPCR Cohort B: To assess histopathological changes in kidney biopsies at Week 12 measured as change from baseline in C3 deposit score Key inclusion criteria Male and female patients 18 years old or older

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?

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...

This trial helped researchers learn how well LNP023 works at different doses and if it is safe to use in people with IgA nephropathy. They learned that protein levels in urine went down more in participants