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 · 5 recorded
| Milestone | Jurisdiction | Brand | Indication | Date | Sentence it was read from |
|---|---|---|---|---|---|
| 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 · a red edge is where a trial was stopped
| 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 | ||||||
| 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 → Oct 2026 expected | 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 | No outcome recorded |
| Phase 2 | NCT04154787 | Nov 2019 → Jan 2023 | idiopathic membranous glomerulonephritis | Novartis Pharmaceuticals | Terminated | No outcome recorded |
| Phase 2 | NCT03896152 | Apr 2019 → Apr 2020 | paroxysmal nocturnal hemoglobinuria | Novartis Pharmaceuticals | Completed | No outcome recorded |
| Phase 2 | NCT03832114 | Feb 2019 → Apr 2021 | glomerulonephritis | Novartis Pharmaceuticals | Completed | No outcome recorded |
| 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 | No outcome recorded |
| Phase 2 | NCT02534909 | Sep 2015 → May 2022 | paroxysmal nocturnal hemoglobinuria | Novartis Pharmaceuticals | Completed | No outcome recorded |
| Phase 315 trials | ||||||
| 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 | No outcome recorded |
| 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 | Active not recruiting | No outcome recorded |
| Phase 3 | NCT04817618 | Jul 2021 → Jan 2027 expected | complement 3 glomerulopathy | Novartis Pharmaceuticals | Recruiting | No outcome recorded |
| 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 | No outcome recorded |
| Phase 3 | NCT04578834 | Jan 2021 → Sep 2025 | IgA glomerulonephritis | Novartis Pharmaceuticals | Completed | No outcome recorded |
| Phase 3 | NCT04558918 | Jan 2021 → Sep 2022 | paroxysmal nocturnal hemoglobinuria | Novartis Pharmaceuticals | Completed | No outcome recorded |
| 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 |
Also used as a comparator or background therapy in 2 trials
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 21 trial | ||||||
| Phase 2 | NCT05268289 Comparator | Aug 2022 → Mar 2026 overdue | lupus nephritis | Novartis Pharmaceuticals | Recruiting | No outcome recorded |
| Phase not stated1 trial | ||||||
| — | NCT05222412 Background | — | complement 3 glomerulopathy, paroxysmal nocturnal hemoglobinuria | Novartis Pharmaceuticals | Available | No outcome recorded |
Press releases naming this drug 31 releases
| Date | Issuer | Release |
|---|---|---|
| 2026-07-17 | Novartis Pharmaceuticals | Novartis Fabhalta® (iptacopan) receives FDA traditional approval as first and only complement inhibitor to significantly slow kidney function decline in primary IgAN novartis.com ↗ |
| 2026-03-29 | Novartis Pharmaceuticals | Novartis IgAN data in New England Journal of Medicine show Fabhalta® slowed kidney function decline by 49.3% novartis.com ↗ |
| 2025-10-16 | Novartis Pharmaceuticals | 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 | Novartis Fabhalta® shows statistically significant and clinically meaningful improvements in hemoglobin in new population of patients with PNH novartis.com ↗ |
| 2025-03-21 | Novartis Pharmaceuticals | 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 | 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 | 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 | Novartis oral Fabhalta® (iptacopan) sustained clinically meaningful results at one year in Phase III C3 glomerulopathy (C3G) trial novartis.com ↗ |
| 2024-08-08 | Novartis Pharmaceuticals | 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 | 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 ↗ |
| 2024-04-15 | Novartis Pharmaceuticals | 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 | 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 | 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 ↗ |
| 2023-12-11 | Novartis Pharmaceuticals | 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 | 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 ↗ |
| 2023-11-20 | Novartis Pharmaceuticals | Novartis showcases significant data updates from Kisqali®, iptacopan and Scemblix® at SABCS and ASH novartis.com ↗ |
| 2023-10-02 | Novartis Pharmaceuticals | Novartis investigational iptacopan Phase III study demonstrates clinically meaningful and highly statistically significant proteinuria reduction in patients with IgA nephropathy (IgAN) novartis.com ↗ |
| 2023-04-26 | Novartis Pharmaceuticals | 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 | 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 | Novartis investigational iptacopan provides clinically meaningful increases in hemoglobin levels in complement-inhibitor-naïve patients with PNH novartis.com ↗ |
| 2022-11-22 | Novartis Pharmaceuticals | Novartis highlights scientific advances with Kisqali, iptacopan, Scemblix and YTB323 data at SABCS and ASH novartis.com ↗ |
| 2022-10-24 | Novartis Pharmaceuticals | 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 | Novartis iptacopan meets primary endpoints in Phase II study in rare kidney disease C3 glomerulopathy (C3G) novartis.com ↗ |
| 2021-06-11 | Novartis Pharmaceuticals | 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 | Novartis announces new interim analysis Phase II data for iptacopan in rare kidney disease C3 glomerulopathy (C3G) novartis.com ↗ |
| 2021-06-06 | Novartis Pharmaceuticals | Novartis announces iptacopan met Phase II study primary endpoint in rare kidney disease IgA nephropathy (IgAN) novartis.com ↗ |
| 2020-12-16 | Novartis Pharmaceuticals | Novartis investigational oral therapy iptacopan (LNP023) receives FDA Breakthrough Therapy Designation for PNH and Rare Pediatric Disease Designation for C3G novartis.com ↗ |
| 2020-10-26 | Novartis Pharmaceuticals | 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 ↗ |
| 2020-10-23 | Novartis Pharmaceuticals | Novartis announces European Medicines Agency (EMA) has granted orphan drug designation for iptacopan (LNP023) in IgA nephropathy (IgAN) novartis.com ↗ |
| 2020-10-09 | Novartis Pharmaceuticals | Novartis received European Medicines Agency (EMA) PRIME designation for iptacopan (LNP) in C3 glomerulopathy (C3G) novartis.com ↗ |
| 2020-08-29 | Novartis Pharmaceuticals | Novartis announces positive results from Phase II study of LNP023 in patients with paroxysmal nocturnal hemoglobinuria (PNH) novartis.com ↗ |
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 ↗ |