Iptacopan
Trade name: Fabhalta
Small moleculeapprovedOrphan Drug FDABreakthrough Therapy FDA
Approved
Dec 5, 2023
Clinical trial activity
28 trials · 2 clinical orgs · 1 marketing orgs
Phase 1
1
Phase 2
25
Phase 3
14
Phase 4
0
Earliest trial started Feb 7, 2018 (NCT03373461)
Timeline
Indications
Studied for
- Agglutination · Phase 2
- Anti-Neutrophil Cytoplasmic Antibody-Associated Vasculitis · Phase 2
- Atypical Hemolytic Uremic Syndrome · Phase 3
- Drugs, Investigational · Phase 3
- Glomerulonephritis · Phase 2
- Glomerulonephritis, IGA · Phase 3
- Glomerulonephritis, Membranoproliferative · Phase 3
- Glomerulonephritis, Membranous · Phase 2
- Hemoglobinuria, Paroxysmal · Phase 3
- Kidney Diseases · Phase 3
- Liver Diseases · Phase 1
- Lupus Nephritis · Phase 2
- Macular Degeneration · Phase 2
- Myasthenia Gravis · Phase 3
- Thrombocytopenia · Phase 2
Mechanism of action
- Complement factor BINHIBITOR
CFB inhibitor
Approval history
- approvedPriority reviewBreakthroughDec 5, 2023
Chemistry & pharmacology
Loading structure…
SMILES
CCO[C@H]1CCN(Cc2c(OC)cc(C)c3[nH]ccc23)[C@H](c2ccc(C(=O)O)cc2)C1- Mol. weight
- 422.53 g/mol
- Lipinski Ro5
- Pass
- Rule of 3
- No
- Chirality
- Single Stereoisomer
- Inorganic
- No
- Polymer
- No
- Delivery
- Oral
- Availability
- Prescription Only
Oral
Yes
Parenteral
No
Topical
No
Black box warningFirst in class
Sources
- ChEMBLCHEMBL5095401 ↗
- ChEMBLCHEMBL4594448 ↗
Clinical trials
| NCT ID | Phase | Start date | Sponsor(s) |
|---|---|---|---|
| NCT06994845 | Phase 3 | Dec 8, 2025 | Novartis |
| NCT06934967 | Phase 3 | Jul 28, 2025 | Novartis |
| NCT06847607 | N/A | Mar 1, 2025 | Chinese Academy of Medical Sciences |
| NCT06797518 | Phase 2 | Feb 24, 2025 | Novartis |
| NCT06388941 | Phase 2 | Aug 5, 2024 | Novartis |
| NCT06517758 | Phase 3 | Jul 31, 2024 | Novartis |
| NCT05935215 | Phase 3 | Mar 29, 2024 | Novartis |
| NCT05795140 | Phase 3 | Feb 12, 2024 | Novartis |
| NCT05755386 | Phase 3 | Oct 2, 2023 | Novartis |
| NCT05630001 | Phase 3 | Apr 28, 2023 | Novartis |
| NCT05268289 | Phase 2 | Aug 10, 2022 | Novartis |
| NCT05230537 | Phase 2 | Feb 17, 2022 | Novartis |
| NCT05086744 | Phase 2 | Nov 29, 2021 | Novartis |
| NCT04889430 | Phase 3 | Nov 8, 2021 | Novartis |
| NCT05078580 | Phase 1 | Oct 29, 2021 | Novartis |
| NCT04817618 | Phase 3 | Jul 28, 2021 | Novartis |
| NCT04747613 | Phase 3 | Jul 27, 2021 | Novartis |
| NCT04820530 | Phase 3 | Jul 19, 2021 | Novartis |
| NCT04557462 | Phase 3 | Feb 17, 2021 | Novartis |
| NCT04578834 | Phase 3 | Jan 27, 2021 | Novartis |
Showing 20 of 28 trials
Page 1 / 2
Organizations
Research & Development (2)
| Organization | Org type | Trials | As lead sponsor | Phases | Earliest year |
|---|---|---|---|---|---|
| Novartis | For profit | 27 | 27 | 4 | 2018 |
| Chinese Academy of Medical Sciences | Academic/Hospital | 1 | 1 | 1 | 2025 |
2 organizations
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