Novartis' First-in-Class Oral Factor B Inhibitor Iptacopan (LNP023) Designated for Breakthrough Therapy in C3 Glomerulopathy

On March 14, the CDE website showed that Novartis' LNP023 capsule is proposed to be included in the breakthrough treatment program for C3 glomerulopathy (C3G). According to the Insight database, LNP023 is currently in the Phase III clinical research stage in China. Phase III clinical trials have been initiated for three indications: C3 glomerulopathy, IgA nephropathy, and paroxysmal nocturnal hemoglobinuria.

Official Website Public Information

Source: CDE Official Website

Iptacopan (LNP023) is a first-in-class, orally administered targeted B-factor inhibitor that targets the alternative complement pathway, a key driver of complement-related renal diseases (CDRDs). At its R&D Day in 2021, Novartis presented the development strategy and subsequent potential milestones for this product. It indicated that Phase 3 clinical data for the PNH indication would be available in 2H 2022, while Phase 3 data for C3G and IgAN are expected in 2023. The first regulatory submission is anticipated to be filed in 2023.

Source: Novartis 2021 R&D Day

Currently, Iptacopan is under development for multiple indications. The development for IgA nephropathy (IgAN), C3 glomerulopathy (C3G), and atypical hemolytic uremic syndrome (aHUS) has entered phase 3 clinical trials. Meanwhile, Novartis is also developing Iptacopan for other indications such as membranous nephropathy (MN) and paroxysmal nocturnal hemoglobinuria (PNH).

Source: Novartis 2021 R&D Day