Iptacopan clinical trials
Iptacopan is named as an intervention in 16 current clinical trials in our China, USA, Germany, Japan, UK, France, Spain, Italy, Turkey, Brazil, Czechia, Israel, Netherlands, Canada, India, Denmark, South Korea, Australia, Switzerland, Belgium, Taiwan, Mexico, Singapore, Poland, Sweden, Norway, Austria and Portugal registers. 11 are recruiting patients now. The most common phase is Phase 3. The sponsor with the most trials is Novartis Pharmaceuticals (14). The most-studied condition is Paroxysmal Nocturnal Hemoglobinuria. The EU clinical trials register (CTIS) separately lists 1 authorised or ongoing trial.
Where Iptacopan is approved
All regulators →Of the three regulators tracked here, the first to approve Iptacopan was the US, on 5 Dec 2023. It is approved in 3 of the 3 regulators tracked here.
From the regulators' own registers: FDA Drugs@FDA, the EMA medicines list and Health Canada's Notice of Compliance database. Dates are the first listed approval of a product containing only this substance; combination products and nationally authorised EU medicines are not counted.
Iptacopan patents and exclusivity
Expiry tracker →Protections still running for marketed products containing Iptacopan, as listed by the regulators. Expired patents and exclusivities, and discontinued products, are not shown.
- 8 listed patents; the last listed expires 15 Jul 2041 (US 12453726).
- Latest patent on the drug substance itself: 25 May 2041 (US 11603363).
- Exclusivity ODE-524: Orphan drug exclusivity: treatment of adults with complement 3 glomerulopathy (c3g), to reduce proteinuria, until 20 Mar 2032.
- Exclusivity ODE-456: Orphan drug exclusivity: treatment of adults with paroxysmal nocturnal hemoglobinuria (pnh), until 5 Dec 2030.
- Exclusivity NCE: New chemical entity, until 5 Dec 2028.
- Exclusivity I-963: New indication: treatment of adults with complement 3 glomerulopathy (c3g), to reduce proteinuria, until 20 Mar 2028.
- Exclusivity I-949: New indication: for the reduction of proteinuria in adults with primary immunoglobulin a nephropathy (igan) at risk of rapid disease progre…, until 7 Aug 2027.
- FABHALTA for treatment of paroxysmal nocturnal haemoglobinuria: until 21 May 2034. Union Register
- FABHALTA for treatment of C3 glomerulopathy: until 1 Apr 2035. Union Register
Reproduced from the FDA Orange Book and Purple Book (checked 8 Nov 2026) and the European Commission's Union Register of medicinal products (CC BY 4.0; the EU expiry date is calculated exactly as the Commission's register displays it). Listed patents and dates are as submitted to or published by the regulator and may not reflect litigation, invalidation, settlements, lapsed fees or later extensions. This is not legal advice or a freedom-to-operate opinion, and it does not say when a generic or biosimilar will launch.
What health systems spend on Iptacopan
All medicines →From NHSBSA Prescription Cost Analysis (Open Government Licence v3.0) and CMS State Drug Utilization Data. Both are gross amounts: neither system publishes its discounts or rebates per drug. Products combining several substances are not counted here.
How Iptacopan works
All drug targets →- Complement factor B inhibitor InhibitorTarget: CFB complement factor B✓ In ChEMBL and the Guide to Pharmacology
- IgA glomerulonephritisPhase 3
- Atypical hemolytic-uremic syndromePhase 3
- Complement 3 glomerulopathyPhase 3
- Immunoglobulin-mediated membranoproliferative glomerulonephritisPhase 3
- Myasthenia gravisPhase 3
- Granulomatosis with polyangiitisPhase 2
- HemolysisPhase 2
- Idiopathic membranous glomerulonephritisPhase 2
- Lupus nephritisPhase 2
- Membranous glomerulonephritisPhase 2
Mechanisms and diseases from ChEMBL via the Open Targets Platform (release 26.09), ChEMBL record CHEMBL4594448, CC BY-SA 3.0. Targets checked against the IUPHAR/BPS Guide to PHARMACOLOGY (2026.3.1), ligand 10710, CC BY-SA 4.0. Trial phases are not approvals: approved uses are shown from the regulators' own records where we hold them.
NICE appraisals of Iptacopan
All NICE appraisals →NICE, which decides whether the NHS in England should fund new medicines, has published 2 technology appraisals of Iptacopan. 2 are current, and 1 of these recommend it for at least some patients.
| Appraisal | NICE recommendation | Published | Status |
|---|---|---|---|
| Iptacopan for treating complement 3 glomerulopathy (terminated appraisal) NICE TA1102 | Terminated (no company submission) | 29 Oct 2025 | Current |
| Iptacopan for treating paroxysmal nocturnal haemoglobinuria NICE TA1000 | Recommended | 4 Sep 2024 | Current |
NICE decisions apply to the NHS in England. In Ireland the HSE decides on reimbursement, advised by the National Centre for Pharmacoeconomics. Titles, categories and dates link to the guidance on nice.org.uk. © NICE 2026 technology appraisal guidance. Available from www.nice.org.uk/guidance. All rights reserved. Subject to Notice of rights. NICE guidance is prepared for the National Health Service in England. All NICE guidance is subject to regular review and may be updated or withdrawn. NICE accepts no responsibility for the use of its content in this product/publication.
Patient leaflets for Iptacopan
The package leaflet is the official guide for patients that comes with every medicine. These links go to the regulators that publish it.
- IEIreland: 1 product on the HPRA listEach product record links to the HPRA, which publishes the leaflet and SmPC.
- EUEuropean Union: FabhaltaEMA product information: package leaflet and summary of product characteristics in every EU language.
- UKUnited Kingdom: Search MHRA ProductsPatient information leaflets and SmPCs for UK-licensed products.
- USUnited States: Search DailyMedFDA-approved labels, including patient and medication guides.
Leaflets differ between brands and countries; always read the one that comes with your own medicine. This is regulatory information, not medical advice.
Trials by status
Trials by phase
Clinical trials of Iptacopan
16 trialsRegisters list studies that are recruiting, about to open, active but closed to enrolment, or enrolling by invitation. The UK and USA registers cover industry-sponsored studies only. Trial titles and statuses are copied from the registry as published.
Iptacopan in the EU trials register (CTIS)
1 trialAuthorised or ongoing trials in the EU Clinical Trials Information System that list a product containing Iptacopan. Many trials are registered in both CTIS and ClinicalTrials.gov, so these are shown separately rather than added to the count above. Trials with a site in Ireland are listed first.
| Study | Phase | Status | Sponsor | Countries |
|---|---|---|---|---|
|
An open-label, single-arm, multicenter roll-over extension program (REP) to characterize the long-term safety and tolerability of iptacopan (LNP023) in patients who have completed a clinical trial (parent study) in the iptacopan pediatric program.
2026-525677-39-00 · CTIS ↗ · Immune System Diseases
|
Phase 3 | Authorised | Novartis Pharma AG | Italy |
Iptacopan across our data
Ireland · EU · USThe same active substance in our other registers, matched by its exact name. Each entry links to the register record it comes from.
Licensed in Ireland (HPRA)
1 single-substance product containing Iptacopan on the HPRA medicines list.
- FABHALTA 200 mg hard capsulesNovartis Europharm Limited · EU/1/24/1802/001-003
Supply shortages
No entry for Iptacopan in the EMA, FDA or German BfArM shortage lists we track.
Conditions studied with Iptacopan
18 conditionsAll 18 conditions
| Condition (as registered) | Trials |
|---|---|
| Paroxysmal Nocturnal Hemoglobinuria | 2 |
| Atypical Hemolytic Uremic Syndrome | 2 |
| Primary IgA Nephropathy | 1 |
| Anti-Neutrophil Cytoplasm Antibodies (ANCA) Associated Vasculitis | 1 |
| C3G | 1 |
| C3 Glomerulopathy | 1 |
| Immune-complex-membranoproliferative Glomerulonephritis | 1 |
| Glomerulonephritis, IGA | 1 |
| Immunoglobulin A Nephropathy | 1 |
| Primary Immunoglobulin A Nephropathy (IgAN) | 1 |
| Paroxysmal Nocturnal Hemoglobinuria (PNH) | 1 |
| Generalized Myasthenia Gravis | 1 |
| IC-MPGN | 1 |
| Thrombotic Microangiopathy | 1 |
| Hematopoietic Stem Cell Transplantation (HSCT) | 1 |
| AIHA - Warm Autoimmune Hemolytic Anemia | 1 |
| AIHA - Cold Autoimmune Hemolytic Anemia | 1 |
| Hemoglobinuria, Paroxysmal | 1 |
Sponsors
lead sponsor per trial| Lead sponsor | Trials |
|---|---|
| Novartis Pharmaceuticals | 14 |
| First Affiliated Hospital of Zhejiang University | 1 |
| Bing Han | 1 |
By country register
A trial with sites in more than one country appears in each of those registers. CTIS does not publish a recruiting flag; its trials may also appear in the registers above.
Related compounds
Questions about Iptacopan trials
How many clinical trials are testing Iptacopan?
16 current trials in our China, USA, Germany, Japan, UK, France, Spain, Italy, Turkey, Brazil, Czechia, Israel, Netherlands, Canada, India, Denmark, South Korea, Australia, Switzerland, Belgium, Taiwan, Mexico, Singapore, Poland, Sweden, Norway, Austria and Portugal registers name Iptacopan (also registered as LNP023) as a drug or biological intervention, as of the registry sync on 10 November 2026. Completed and withdrawn studies are not counted.
Are Iptacopan trials recruiting patients?
Yes. 11 of the 16 trials are recruiting: 8 with sites in China, 7 with sites in USA, 6 with sites in Germany, 7 with sites in Japan, 5 with sites in UK, 5 with sites in France, 5 with sites in Spain, 6 with sites in Italy, 6 with sites in Turkey, 6 with sites in Brazil, 5 with sites in Czechia, 6 with sites in Israel, 5 with sites in Netherlands, 4 with sites in Canada, 4 with sites in India, 2 with sites in Denmark, 2 with sites in South Korea, 2 with sites in Australia, 3 with sites in Switzerland, 2 with sites in Belgium, 2 with sites in Taiwan, 2 with sites in Mexico, 1 with sites in Singapore, 1 with sites in Poland, 1 with sites in Sweden and 1 with sites in Norway. Eligibility and site contacts are on each trial's record. Only the study team can confirm whether a patient is eligible.
Who sponsors Iptacopan clinical trials?
Novartis Pharmaceuticals (14), First Affiliated Hospital of Zhejiang University (1) and Bing Han (1). The number in brackets is how many trials each organisation leads.
Is Iptacopan licensed in Ireland?
Yes. The HPRA medicines list includes 1 single-substance product containing Iptacopan, for example FABHALTA 200 mg hard capsules (Novartis Europharm Limited). A licence does not mean a product is currently marketed or reimbursed; see each product's record.
Is there a Iptacopan shortage?
Not in the lists we track: Iptacopan has no entry in the EMA, FDA or German BfArM shortage registers as of their latest sync.
Where does this information come from?
Every trial, status, phase and sponsor on this page is copied from public ClinicalTrials.gov records by a daily sync. Nothing here describes how Iptacopan works or whether it is effective, and it is not medical advice.
How this page is built
Everything on this page is copied from public ClinicalTrials.gov registry records — it is not written from memory, and nothing here describes how Iptacopan works or whether it is effective. Each trial links to its own record so you can check it yourself.
- ClinicalTrials.gov (U.S. National Library of Medicine) ↗ Trial IDs, titles, intervention names and synonyms, phase, overall status, lead sponsor and conditions for every trial on this page
This page is built automatically: a daily sync job reads the registry records behind our country trial registers, collects every trial that lists Iptacopan (or a registered synonym) as a drug or biological intervention, and tabulates the registry fields directly. Placebos and generic comparators are excluded. There is no AI drafting and no per-compound editorial pass, and the page has not been clinically reviewed. The “last verified” date above is the timestamp of that sync, not a review date. Our general editorial approach is described in our editorial standards and corrections policy. The ClinicalTrials.gov data on this page was last retrieved on 10 Nov 2026. How every register is built: methodology · fixes we have made: corrections log.