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
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
Phase 3: Anemia|Atypical Hemolytic Uremic Syndrome|Azotemia|Complement 3 Glomerulopathy|Glomerulonephritis|Glomerulonephritis, Membranoproliferative|Hemolytic-Uremic Syndrome|IgA Nephropathy|Kidney Failure, Chronic|Myasthenia Gravis|Paroxysmal Hemoglobinuria|Paroxysmal Nocturnal Hemoglobinuria|Proteinuria|Thrombotic Microangiopathies
Phase 2: ANCA Vasculitis|Granuloma|Kidney Diseases|Lupus Erythematosus, Systemic|Lupus Nephritis|Macular Degeneration|Microscopic Polyangiitis
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 |
