Drugs / Iptacopan
Iptacopan
also known as
LNP023 · COD41 · Fabhalta · IPTACOPAN HYDROCHLORIDE · Iptacopan hydrochloride monohydrate · LNP-023 HYDROCHLORIDE MONOHYDRATE · LNP-023-AAB · NVP-LNP023 · +2 more
Small molecule targets CFB via inhibition
Regulatory milestones approvals, filings & regulatory actions · 9 recorded
| Milestone | Jurisdiction | Brand | Indication | Date | Sentence it was read from |
|---|---|---|---|---|---|
| Filed C3 glomerulonephritis 1 milestone | |||||
| Filed | US (FDA) | Fabhalta | C3 glomerulonephritis | 2025-01-31 | “Submission for the treatment of C3 glomerulopathy (C3G) was completed in the US, and the FDA granted Priority Review status to Fabhalta in this indication.” novartis.com ↗ |
| Filed IgA glomerulonephritis 1 milestone | |||||
| Filed | US (FDA) | Fabhalta | IgA glomerulonephritis | 2024-04-23 | “Fabhalta (iptacopan) FDA filing accepted for IgAN” novartis.com ↗ |
| Filed paroxysmal nocturnal hemoglobinuria 2 milestones | |||||
| Filed | US (FDA) | — | paroxysmal nocturnal hemoglobinuria | — | “Iptacopan PNH – regulatory submissions completed in the US and Europe” novartis.com ↗ |
| Filed | EU (EMA) | — | paroxysmal nocturnal hemoglobinuria | — | “Iptacopan PNH – regulatory submissions completed in the US and Europe” novartis.com ↗ |
| Approved Indication not stated 3 milestones | |||||
| Label expansions (3) | US (FDA) | FABHALTA | — | 2024-08-07 – 2026-07-16 | fda.gov ↗ |
| Approved | EU (EMA) | Fabhalta | — | 2024-05-17 | europa.eu ↗ |
| Approved | US (FDA) | FABHALTA | — | 2023-12-05 | fda.gov ↗ |
Trials 28
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 11 trial | ||||||
| Phase 1 | NCT05078580 | Nov 2021 → Jun 2022 | — | Novartis Pharmaceuticals | Completed | No outcome recorded |
| Phase 210 trials · 3 met primary · 1 missed | ||||||
| Phase 2 | NCT06797518 | Feb 2025 → Nov 2027 expected | IgA glomerulonephritis | Novartis Pharmaceuticals | Recruiting | No outcome recorded |
| Phase 2 | NCT06388941 | Aug 2024 → Dec 2026 expected | anti-neutrophil cytoplasmic antibody-associated vasculitis | Novartis Pharmaceuticals | Active not recruiting | No outcome recorded |
| Phase 2 | NCT05230537 | Feb 2022 → Sep 2026 overdue | age-related macular degeneration | Novartis Pharmaceuticals | Active not recruiting | No outcome recorded |
| Phase 2 | NCT05086744 | Dec 2021 → Sep 2023 | autoimmune thrombocytopenia, cold agglutinin disease | Novartis Pharmaceuticals | Terminated | Mixed Stop: Business |
| Phase 2 | NCT04154787 | Nov 2019 → Jan 2023 | idiopathic membranous glomerulonephritis | Novartis Pharmaceuticals | Terminated | Missed primary Stop: Business |
| Phase 2 | NCT03896152 | Apr 2019 → Apr 2020 | paroxysmal nocturnal hemoglobinuria | Novartis Pharmaceuticals | Completed | Met primary |
| Phase 2 | NCT03832114 | Feb 2019 → Apr 2021 | glomerulonephritis | Novartis Pharmaceuticals | Completed | Met primary |
| Phase 2 | NCT03439839 | Apr 2018 → Apr 2020 | paroxysmal nocturnal hemoglobinuria | Novartis Pharmaceuticals | Completed | No outcome recorded |
| Phase 2 | NCT03373461 | Feb 2018 → Dec 2020 | IgA glomerulonephritis | Novartis Pharmaceuticals | Completed | Met primary |
| Phase 2 | NCT02534909 | Sep 2015 → May 2022 | paroxysmal nocturnal hemoglobinuria | Novartis Pharmaceuticals | Completed | No outcome recorded |
| Phase 315 trials · 5 met primary | ||||||
| Phase 3 | NCT06994845 | Nov 2025 → Jun 2030 expected | IgA glomerulonephritis | Novartis Pharmaceuticals | Recruiting | No outcome recorded |
| Phase 3 | NCT06934967 | Oct 2025 → Nov 2031 expected | paroxysmal nocturnal hemoglobinuria | Novartis Pharmaceuticals | Recruiting | No outcome recorded |
| Phase 3 | NCT06517758 | Jul 2024 → Apr 2027 expected | generalized myasthenia gravis | Novartis Pharmaceuticals | Recruiting | No outcome recorded |
| Phase 3 | NCT05795140 | May 2024 → Jul 2032 expected | atypical hemolytic-uremic syndrome | Novartis Pharmaceuticals | Recruiting | No outcome recorded |
| Phase 3 | NCT05935215 | Feb 2024 → Jul 2028 expected | atypical hemolytic-uremic syndrome | Novartis Pharmaceuticals | Recruiting | No outcome recorded |
| Phase 3 | NCT05755386 | Oct 2023 → Oct 2028 expected | primary membranoproliferative glomerulonephritis | Novartis Pharmaceuticals | Recruiting | No outcome recorded |
| Phase 3 | NCT05630001 | Apr 2023 → Oct 2024 | paroxysmal nocturnal hemoglobinuria | Novartis Pharmaceuticals | Completed | Met primary |
| Phase 3 | NCT04889430 | Jan 2022 → Apr 2026 | atypical hemolytic-uremic syndrome | Novartis Pharmaceuticals | Completed | No outcome recorded |
| Phase 3 | NCT04557462 | Sep 2021 → Dec 2030 expected | IgA glomerulonephritis | Novartis Pharmaceuticals | Recruiting | No outcome recorded |
| Phase 3 | NCT04817618 | Jul 2021 → Jan 2027 expected | complement 3 glomerulopathy | Novartis Pharmaceuticals | Recruiting | Met primary |
| Phase 3 | NCT04747613 | Jul 2021 → Oct 2027 expected | paroxysmal nocturnal hemoglobinuria | Novartis Pharmaceuticals | Active not recruiting | No outcome recorded |
| Phase 3 | NCT04820530 | Jul 2021 → Nov 2022 | paroxysmal nocturnal hemoglobinuria | Novartis Pharmaceuticals | Completed | Met primary |
| Phase 3 | NCT04558918 | Jan 2021 → Sep 2022 | paroxysmal nocturnal hemoglobinuria | Novartis Pharmaceuticals | Completed | Met primary |
| Phase 3 | NCT04578834 | Jan 2021 → Sep 2025 | IgA glomerulonephritis | Novartis Pharmaceuticals | Completed | Met primary |
| Phase 3 | NCT03955445 | Oct 2019 → May 2036 expected | complement 3 glomerulopathy, immunoglobulin-mediated membranoproliferative glomerulonephritis | Novartis Pharmaceuticals | Recruiting | No outcome recorded |
| Phase not applicable2 trials | ||||||
| — | NCT07347990 | Jan 2026 → Dec 2027 expected | disease related to hematopoietic stem cell transplant, thrombotic microangiopathy | First Affiliated Hospital of Zhejiang University | Not yet recruiting | No outcome recorded |
| — | NCT06847607 | Mar → Oct 2025 overdue | autoimmune hemolytic anemia, cold type, autoimmune hemolytic anemia, warm type | Bing Han | Not yet recruiting | No outcome recorded |
| Comparator or background therapy2 trials | ||||||
| Phase 2 | NCT05268289 Comparator | Aug 2022 → Mar 2026 overdue | lupus nephritis | Novartis Pharmaceuticals | Recruiting | No outcome recorded |
| — | NCT05222412 Background | — | complement 3 glomerulopathy, paroxysmal nocturnal hemoglobinuria | Novartis Pharmaceuticals | Available | No outcome recorded |
News releases announcing trial results or a regulatory action · 37
| Date | Issuer | Release |
|---|---|---|
| 2026-07-17 | Novartis Pharmaceuticals | Regulatory Novartis Fabhalta® (iptacopan) receives FDA traditional approval as first and only complement inhibitor to significantly slow kidney function decline in primary IgAN novartis.com ↗
Basel, July 17, 2026 – Novartis today announced that the US Food and Drug Administration (FDA) has granted traditional approval for Fabhalta ® (iptacopan) to slow kidney function decline in adults with primary immunoglobulin A nephropathy (IgAN) at risk of disease progression 1 . |
| 2026-05-26 | Novartis Pharmaceuticals | Results New Novartis data at ERA 2026 advance scientific understanding of kidney disease and reinforce portfolio strength novartis.com ↗
Iptacopan achieves near-normal kidney function decline in prespecified IgA nephropathy (IgAN) patient subgroups: APPLAUSE-IgAN final data |
| 2026-03-29 | Novartis Pharmaceuticals | Results Novartis IgAN data in New England Journal of Medicine show Fabhalta® slowed kidney function decline by 49.3% novartis.com ↗
Novartis today announced final two-year results from the Phase III APPLAUSE‑IgAN study of Fabhalta ® (iptacopan) in IgA nephropathy (IgAN). |
| 2025-11-25 | Novartis Pharmaceuticals | Results Novartis data underscore pioneering scientific innovation in hematology and oncology at ASH and SABCS novartis.com ↗
Oral iptacopan monotherapy demonstrates clinically meaningful hemoglobin increases in patients with paroxysmal nocturnal hemoglobinuria with baseline hemoglobin levels 10 to <12 g/dL on anti-C5 therapy: Subgroup analysis of the APPULSE-PNH Phase 3b trial |
| 2025-10-16 | Novartis Pharmaceuticals | Results Novartis Fabhalta® (iptacopan) meets Phase III primary endpoint, slows kidney function decline in patients with IgA nephropathy (IgAN) novartis.com ↗ |
| 2025-06-12 | Novartis Pharmaceuticals | Results Novartis Fabhalta® shows statistically significant and clinically meaningful improvements in hemoglobin in new population of patients with PNH novartis.com ↗
Novartis announced positive results from APPULSE-PNH, a Phase IIIB study evaluating the efficacy and safety of twice-daily oral monotherapy Fabhalta ® (iptacopan) in adult patients with paroxysmal nocturnal hemoglobinuria (PNH) with Hb levels ≥10g/dL who switched from anti-C5 therapies (eculizumab or ravulizumab) 1 . |
| 2025-05-15 | Novartis Pharmaceuticals | Results New Novartis data at ASCO and EHA showcase momentum of pioneering portfolio with promising pipeline novartis.com ↗
Oral iptacopan monotherapy demonstrates clinically meaningful hemoglobin (Hb) increases in patients (pts) with paroxysmal nocturnal hemoglobinuria (PNH) and Hb ≥10 g/dL on anti-C5 therapy |
| 2025-03-21 | Novartis Pharmaceuticals | Regulatory Novartis receives third FDA approval for oral Fabhalta® (iptacopan) – the first and only treatment approved in C3 glomerulopathy (C3G) novartis.com ↗ |
| 2025-02-28 | Novartis Pharmaceuticals | Regulatory Novartis oral Fabhalta® (iptacopan) receives positive CHMP opinion for the treatment of adults living with C3 glomerulopathy (C3G) novartis.com ↗ |
| 2024-12-06 | Novartis Pharmaceuticals | Results New Phase IIIB data shows Novartis Fabhalta® improved hemoglobin levels in adult patients with paroxysmal nocturnal hemoglobinuria who switched from anti-C5 therapy novartis.com ↗ |
| 2024-10-27 | Novartis Pharmaceuticals | Results Novartis oral Fabhalta® (iptacopan) sustained clinically meaningful results at one year in Phase III C3 glomerulopathy (C3G) trial novartis.com ↗
These data confirm treatment with Fabhalta resulted in clinically meaningful proteinuria reduction, which was seen as early as 14 days, and sustained at 12 months. |
| 2024-08-08 | Novartis Pharmaceuticals | Regulatory Novartis receives FDA accelerated approval for Fabhalta® (iptacopan), the first and only complement inhibitor for the reduction of proteinuria in primary IgA nephropathy (IgAN) novartis.com ↗ |
| 2024-05-25 | Novartis Pharmaceuticals | Results Novartis presents latest Phase III Fabhalta® (iptacopan) data in C3 glomerulopathy (C3G) showing clinically meaningful and statistically significant 35.1% proteinuria reduction vs. placebo novartis.com ↗ |
| 2024-05-15 | Novartis Pharmaceuticals | Results Novartis highlights pioneering innovation in CML with data from Scemblix® Phase III ASC4FIRST study in newly diagnosed patients at ASCO and EHA novartis.com ↗
Effects of oral iptacopan monotherapy, including increased paroxysmal nocturnal hemoglobinuria red blood cell clone size, are sustained in anti-c5-treated patients with anemia: final APPLY-PNH data |
| 2024-04-15 | Novartis Pharmaceuticals | Results New Novartis Fabhalta® (iptacopan) data show clinically meaningful and statistically significant proteinuria reduction of 38.3% versus placebo for patients with IgA nephropathy (IgAN) novartis.com ↗ |
| 2024-03-22 | Novartis Pharmaceuticals | Regulatory Novartis Fabhalta® (iptacopan) receives positive CHMP opinion as first oral monotherapy for adult patients with paroxysmal nocturnal hemoglobinuria (PNH) novartis.com ↗ |
| 2023-12-11 | Novartis Pharmaceuticals | Results Novartis presents new 48-week results from Phase III APPLY-PNH trial showing sustained efficacy and long-term safety of Fabhalta® (iptacopan) in adults with paroxysmal nocturnal hemoglobinuria (PNH) novartis.com ↗
Fabhalta had a similar safety profile at 48 weeks vs. 24 weeks 1,2 . |
| 2023-12-11 | Novartis Pharmaceuticals | Results Novartis investigational iptacopan Phase III study demonstrates clinically meaningful and statistically significant proteinuria reduction in patients with C3 glomerulopathy (C3G) novartis.com ↗ |
| 2023-12-06 | Novartis Pharmaceuticals | Regulatory Novartis receives FDA approval for Fabhalta® (iptacopan), offering superior hemoglobin improvement in the absence of transfusions as the first oral monotherapy for adults with PNH novartis.com ↗
Novartis today announced that the U.S. Food and Drug Administration (FDA) approved Fabhalta ® (iptacopan) as the first oral monotherapy for the treatment of adults with paroxysmal nocturnal hemoglobinuria (PNH) 1 . |
| 2023-11-20 | Novartis Pharmaceuticals | Results Novartis showcases significant data updates from Kisqali®, iptacopan and Scemblix® at SABCS and ASH novartis.com ↗
Clinical Breakthrough Hemolysis (BTH) during Monotherapy with the Oral Factor B Inhibitor Iptacopan Is Generally Not Severe and Managed without Treatment Discontinuation |
| 2023-10-02 | Novartis Pharmaceuticals | Results Novartis investigational iptacopan Phase III study demonstrates clinically meaningful and highly statistically significant proteinuria reduction in patients with IgA nephropathy (IgAN) novartis.com ↗
Phase III APPLAUSE-IgAN study met its pre-specified interim analysis primary endpoint, demonstrating superiority of iptacopan vs placebo in proteinuria reduction 1 |
| 2023-05-11 | Novartis Pharmaceuticals | Results Novartis to feature new PNH and CML scientific data from broad hematology portfolio at European Hematology Association Annual Meeting novartis.com ↗
Updated iptacopan data confirm hemolysis control in paroxysmal nocturnal hemoglobinuria (PNH) patients from Phase III APPLY-PNH and APPOINT-PNH studies |
| 2023-04-26 | Novartis Pharmaceuticals | Results 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 novartis.com ↗ |
| 2022-12-13 | Novartis Pharmaceuticals | Results Novartis presents pivotal Phase III APPLY-PNH data at ASH demonstrating investigational oral monotherapy iptacopan superiority over anti-C5 novartis.com ↗ |
| 2022-12-08 | Novartis Pharmaceuticals | Results Novartis investigational iptacopan provides clinically meaningful increases in hemoglobin levels in complement-inhibitor-naïve patients with PNH novartis.com ↗ |
| 2022-11-22 | Novartis Pharmaceuticals | Results Novartis highlights scientific advances with Kisqali, iptacopan, Scemblix and YTB323 data at SABCS and ASH novartis.com ↗
Oral monotherapy with iptacopan, a proximal complement inhibitor of factor B, has superior efficacy to intravenous terminal complement inhibition with standard of care Eculizumab or Ravulizumab and favorable safety in patients with paroxysmal nocturnal hemoglobinuria and residual anemia |
| 2022-10-25 | Novartis Pharmaceuticals | Results Novartis maintains growth momentum and confirms FY’22 Group guidance novartis.com ↗
In October, Ph3 APPLY-PNH trial met its two primary endpoints for superiority vs anti-C5 treatment in adult paroxysmal nocturnal hemoglobinuria (PNH) patients with residual anemia despite prior anti-C5 treatment |
| 2022-10-24 | Novartis Pharmaceuticals | Results Novartis investigational oral monotherapy iptacopan demonstrates clinically meaningful superiority over anti-C5 treatment in Phase III APPLY-PNH study novartis.com ↗ |
| 2021-11-04 | Novartis Pharmaceuticals | Results Novartis iptacopan meets primary endpoints in Phase II study in rare kidney disease C3 glomerulopathy (C3G) novartis.com ↗
Novartis today announced that a Phase II study of investigational iptacopan (LNP023) – a first-in-class, oral, selective factor B inhibitor – in patients with C3 glomerulopathy (C3G) met primary endpoints in both patient cohorts 1 . |
| 2021-06-11 | Novartis Pharmaceuticals | Results Novartis investigational oral therapy iptacopan (LNP023) shows benefit as monotherapy in treatment-naïve patients with rare and life-threatening blood disorder paroxysmal nocturnal hemoglobinuria novartis.com ↗ |
| 2021-06-07 | Novartis Pharmaceuticals | Results Novartis announces new interim analysis Phase II data for iptacopan in rare kidney disease C3 glomerulopathy (C3G) novartis.com ↗ |
| 2021-06-06 | Novartis Pharmaceuticals | Results Novartis announces iptacopan met Phase II study primary endpoint in rare kidney disease IgA nephropathy (IgAN) novartis.com ↗ |
| 2020-12-16 | Novartis Pharmaceuticals | Regulatory Novartis investigational oral therapy iptacopan (LNP023) receives FDA Breakthrough Therapy Designation for PNH and Rare Pediatric Disease Designation for C3G novartis.com ↗
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). |
| 2020-10-26 | Novartis Pharmaceuticals | Results Novartis presents promising interim Phase II data of potential first-in-class oral therapy iptacopan (LNP023) in rare renal disease C3 glomerulopathy (C3G) novartis.com ↗
Novartis today announced positive Phase II interim analysis results for iptacopan (LNP023), an investigational oral treatment for C3 glomerulopathy (C3G), presented at the virtually held American Society of Nephrology (ASN) 2020 Annual Meeting. |
| 2020-10-23 | Novartis Pharmaceuticals | Regulatory Novartis announces European Medicines Agency (EMA) has granted orphan drug designation for iptacopan (LNP023) in IgA nephropathy (IgAN) novartis.com ↗
Novartis today announced that the European Medicines Agency (EMA) has granted an orphan drug designation for iptacopan (LNP023) in IgA nephropathy (IgAN), following a recommendation from the Committee for Orphan Medicinal Products (COMP). |
| 2020-10-09 | Novartis Pharmaceuticals | Regulatory Novartis received European Medicines Agency (EMA) PRIME designation for iptacopan (LNP) in C3 glomerulopathy (C3G) novartis.com ↗
The European Medicines Agency has granted iptacopan a priority medicines (PRIME) designation in C3 glomerulopathy ( C3G ) . |
| 2020-08-29 | Novartis Pharmaceuticals | Results Novartis announces positive results from Phase II study of LNP023 in patients with paroxysmal nocturnal hemoglobinuria (PNH) novartis.com ↗
In the open-label Phase II study (NCT03439839), LNP023 demonstrated improvements in hematological response and biomarkers of disease activity in patients with active hemolysis despite treatment with eculizumab. |
All press releases naming this drug 37 releases
Evidence & citations 15 cited values
Every value below carries the sentence it was read from. 32 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | iptacopan | “Iptacopan (LNP023) is a novel, oral selective inhibitor of complement factor B under clinical development for paroxysmal nocturnal hemoglobinuria (PNH).” PMID 35561315 ↗ Aug 202222“Evaluate Long-term Safety, Tolerability and Efficacy of Iptacopan in Study Participants With aHUS” NCT05795140 ↗ “Iptacopan 200mg b.i.d. hard gelatin capsule. After 24 weeks of LNP023 200mg b.i.d. treatment” NCT04558918 ↗ “Efficacy and Safety of Iptacopan in Patients With C3 Glomerulopathy.” PMID 38106570 ↗ Sep 2023 “Drug: Iptacopan” NCT06797518 ↗ |
| Known as | COD41 | ChEMBL registry synonym — accepted as the source's own label CHEMBL4594448 ↗ |
| Known as | Fabhalta | ChEMBL registry synonym — accepted as the source's own label CHEMBL4594448 ↗ |
| Known as | IPTACOPAN HYDROCHLORIDE | ChEMBL registry synonym — accepted as the source's own label CHEMBL4594448 ↗ |
| Known as | Iptacopan hydrochloride monohydrate | ChEMBL registry synonym — accepted as the source's own label CHEMBL4594448 ↗ |
| Known as | LNP-023 HYDROCHLORIDE MONOHYDRATE | ChEMBL registry synonym — accepted as the source's own label CHEMBL4594448 ↗ |
| Known as | LNP-023-AAB | ChEMBL registry synonym — accepted as the source's own label CHEMBL4594448 ↗ |
| Known as | LNP023 | “A Phase 1, Single Dose, Open-label Study to Investigate the Pharmacokinetics and Safety of Iptacopan (LNP023) in Participants With Mild, Moderate, and Severe Hepatic Impairment...” NCT05078580 ↗12“Iptacopan (LNP023) is a novel, oral selective inhibitor of complement factor B under clinical development for paroxysmal nocturnal hemoglobinuria (PNH).” PMID 35561315 ↗ Aug 2022 “evaluate the efficacy and safety of iptacopan (LNP023) in idiopathic immune complex mediated membranoproliferative glomerulonephritis” NCT05755386 ↗ “Iptacopan (LNP023) oral capsules” NCT05230537 ↗ “Iptacopan (LNP023)” NCT04820530 ↗ |
| Known as | NVP-LNP023 | ChEMBL registry synonym — accepted as the source's own label CHEMBL4594448 ↗ |
| Known as | NVP-LNP023-AAB | ChEMBL registry synonym — accepted as the source's own label CHEMBL4594448 ↗ |
| Known as | NVP-LNP023-NX | ChEMBL registry synonym — accepted as the source's own label CHEMBL4594448 ↗ |
| Action | Inhibit | “Iptacopan (LNP023) is a novel, oral selective inhibitor of complement factor B” PMID 35561315 ↗ Aug 20221“Complement factor B inhibitor” CHEMBL4594448 ↗ |
| Modality | Small molecule | “LNP023 is a novel oral small molecular weight compound” NCT03896152 ↗ |
| Route | Oral | “LNP023 at a dose of 200 mg administered orally twice daily (b.i.d.) for approximately 20 weeks” NCT02534909 ↗ |
| Target | CFB | “Complement factor B inhibitor” CHEMBL4594448 ↗ |