Ozmosi | Iptacopan Drug Profile

Product Description

Iptacopan is an investigational first-in-class, orally administered targeted factor B inhibitor of the alternative complement pathway. It acts upstream of the C5 terminal pathway, preventing not only intravascular but also extravascular hemolysis in PNH. In doing so, iptacopan targets a key part of the biology responsible for PNH while offering an oral monotherapy option. Iptacopan is currently in development for a number of other complement-mediated diseases (CMDs) where significant unmet needs exist. (Sourced from: https://www.novartis.com/news/media-releases/novartis-investigational-iptacopan-provides-clinically-meaningful-increases-hemoglobin-levels-complement-inhibitor-naive-patients-pnh)

Mechanisms of Action: CFB Inhibitor

Novel Mechanism: No

Modality: Small Molecule

Route of Administration: Oral

FDA Designation: Accelerated Approval - IgA Nephropathy|Proteinuria *

Approval Status: Approved

Approved Countries: None

Approved Indications: None

Known Adverse Events: None

Company: Novartis
Company Location: Europe
Company CEO: Vasant Narasimhan
Additional Commercial Interests: None

Clinical Description

Map of Global Clinical Trials for Iptacopan

Countries in Clinic: Argentina, Australia, Austria, Belgium, Brazil, Canada, Chile, China, Colombia, Czech Republic, Denmark, Finland, France, Germany, Greece, Guatemala, Hong Kong, Hungary, India, Israel, Italy, Japan, Korea, Lithuania, Malaysia, Mexico, Netherlands, Norway, Philippines, Poland, Portugal, Puerto Rico, Russia, Singapore, Slovakia, Slovenia, South Africa, South Korea, Spain, Sweden, Switzerland, Taiwan, Thailand, Turkey, United Kingdom, United States, Unknown Location, Vietnam

Active Clinical Trial Count:

Recent & Upcoming Milestones

Clinical Outcomes Reported - Novartis presented P3 Paroxysmal Nocturnal Hemoglobinuria results on 2025-12-08 for Iptacopan
Clinical Outcomes Reported - Novartis presented P3 IgA Nephropathy results on 2025-11-07 for Iptacopan
Clinical Outcomes Reported - Novartis presented P3 IgA Nephropathy results on 2025-10-16 for Iptacopan

Highest Development Phases

Trial ID	Trial	Phase	Trial Status	Disease	Primary Completion Date	Probability of Success	Latest Trial Update Date	Data Updated
NCT06797518	CLNP023A2201	P2	Recruiting	Glomerulonephritis\|IgA Nephropathy\|Kidney Diseases	2027-11-27	12%	2025-07-08	Primary Completion Date\|Primary Endpoints\|Start Date\|Study Completion Date
NCT06388941	CLNP023R12201	P2	Recruiting	ANCA Vasculitis	2027-09-09	2%	2024-10-09	Primary Endpoints
NCT05230537	CLNP023E12201	P2	Active, not recruiting	Macular Degeneration	2026-10-01	50%	2025-07-04	Primary Completion Date\|Primary Endpoints
NCT05268289	CLNP023K12201	P2	Recruiting	Lupus Erythematosus, Systemic\|Lupus Nephritis\|Proteinuria	2026-03-30	12%	2025-12-23
NCT04557462	CLNP023A2002B	P3	Recruiting	Glomerulonephritis\|Kidney Failure, Chronic\|IgA Nephropathy	2032-10-25	54%	2025-12-25	Primary Endpoints
NCT06934967	CLNP023I12201	P3	Recruiting	Paroxysmal Nocturnal Hemoglobinuria\|Paroxysmal Hemoglobinuria\|Anemia	2031-11-19	51%	2025-12-06	Primary Endpoints
NCT05755386	APPARENT	P3	Recruiting	Glomerulonephritis, Membranoproliferative	2028-10-27	53%	2025-07-11	Primary Completion Date\|Primary Endpoints\|Study Completion Date\|Treatments
NCT04889430	APPELHUS	P3	Active, not recruiting	Atypical Hemolytic Uremic Syndrome\|Thrombotic Microangiopathies	2028-09-19	52%	2025-12-12	Patient Enrollment\|Primary Endpoints\|Trial Status
NCT05935215	CLNP023F12302	P3	Recruiting	Atypical Hemolytic Uremic Syndrome\|Azotemia\|Hemolytic-Uremic Syndrome	2028-07-21	27%	2024-10-02	Primary Completion Date\|Primary Endpoints\|Study Completion Date\|Treatments
NCT04747613	CLNP023C12001B	P3	Active, not recruiting	Paroxysmal Hemoglobinuria\|Paroxysmal Nocturnal Hemoglobinuria	2027-10-19	53%	2025-02-08	Patient Enrollment\|Primary Endpoints\|Treatments\|Trial Status
NCT06517758	CLNP023Q12301	P3	Recruiting	Myasthenia Gravis	2026-12-28	9%	2024-07-25	Primary Endpoints\|Treatments
NCT04817618	APPEAR-C3G	P3	Recruiting	Complement 3 Glomerulopathy	2026-03-27	52%	2024-05-25	Primary Endpoints
2023-508840-22-00	CLNP023F12301	P3	Recruiting	Atypical Hemolytic Uremic Syndrome	2025-12-15		2025-05-02	Treatments
NCT04578834	APPLAUSE-IgAN	P3	Completed	IgA Nephropathy\|Proteinuria\|Glomerulonephritis	2025-09-19	53%	2025-10-09	Primary Completion Date\|Primary Endpoints\|Study Completion Date\|Treatments\|Trial Status
2022-502160-20-00	CLNP023B12302	P3	Recruiting	Glomerulonephritis, Membranoproliferative	2028-05-31		2025-05-02	Treatments
2023-509332-26-00	CLNP023K12201	P2	Recruiting	Lupus Nephritis	2028-09-28		2025-05-02	Treatments
NCT03955445	CLNP023B12001B	P3	Recruiting	Glomerulonephritis, Membranoproliferative	2036-05-30	54%	2025-04-02	Patient Enrollment\|Primary Completion Date\|Primary Endpoints\|Study Completion Date\|Treatments
NCT05795140	CLNP023F12001B	P3	Recruiting	Thrombotic Microangiopathies\|Atypical Hemolytic Uremic Syndrome\|Glomerulonephritis, Membranoproliferative	2032-12-23	53%	2025-08-27	Primary Endpoints
2023-507064-39-00	CLNP023Q12301	P3	Recruiting	Myasthenia Gravis	2032-04-12		2025-05-02	Treatments
2024-515926-10-00	CLNP023I12201	P3	Not yet recruiting	Paroxysmal Hemoglobinuria\|Paroxysmal Nocturnal Hemoglobinuria	2031-10-31
jRCT2031250555	jRCT2031250555	P3	Recruiting	IgA Nephropathy	2030-09-30
NCT06994845	CLNP023G12301	P3	Recruiting	IgA Nephropathy\|Proteinuria\|Glomerulonephritis	2030-06-28	10%	2025-12-11	Patient Enrollment\|Primary Endpoints\|Treatments
2023-504550-35-00	CLNP023F12302	P3	Recruiting	Atypical Hemolytic Uremic Syndrome	2029-07-19		2025-05-02	Treatments
2022-502965-34-00	CLNP023F12001B	P3	Recruiting	Atypical Hemolytic Uremic Syndrome	2029-04-15		2025-05-02	Treatments
jRCT2031230686	jRCT2031230686	P3	Not yet recruiting	Atypical Hemolytic Uremic Syndrome	2029-01-31

Recent News Events

Date	Type	Title
11/25/2025	News Article	Novartis data underscore pioneering scientific innovation in Hematology and Oncology at ASH and SABCS
11/20/2025	News Article	Novartis projects +5-6% cc sales CAGR 2025-2030, with long-term growth backed by 30+ potential high-value pipeline assets
11/19/2025	News Article	Spherix Global Insights Reports Rapidly Evolving U.S. PNH Market as Ultomiris Prescriptions Reach Parity with Iron Supplements
11/04/2025	News Article	New Novartis data at ASN Kidney Week and AHA Scientific Sessions demonstrate momentum of broad CRM portfolio and pipeline

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Iptacopan

Alternative Names: iptacopan, lnp-023, lnp023, lnp 023, FABHALTA Clinical Status: Active Latest Update: 2026-01-09 Latest Update Note: Clinical Trial Update