Drug Type Small molecule drug |
Synonyms Iptacopan Hydrochloride, 伊普可泮, LNP 023 + [6] |
Target |
Action inhibitors |
Mechanism CFB inhibitors(Complement factor B inhibitors) |
Therapeutic Areas |
Active Indication |
Inactive Indication |
Originator Organization |
Inactive Organization- |
License Organization- |
Drug Highest PhaseApproved |
First Approval Date United States (05 Dec 2023), |
RegulationPriority Review (United States), Breakthrough Therapy (United States), Accelerated Approval (United States), Orphan Drug (United States), Orphan Drug (European Union), PRIME (European Union), Breakthrough Therapy (China), Orphan Drug (South Korea), Orphan Drug (Australia), Priority Review (Australia), Rare Pediatric Disease (United States), Priority Review (China) |
Molecular FormulaC25H33ClN2O5 |
InChIKeyJUWBBUFSAGEROP-VVJLZRNGSA-N |
CAS Registry2447007-60-3 |
Indication | Country/Location | Organization | Date |
---|---|---|---|
C3 glomerulopathy | United States | 20 Mar 2025 | |
Glomerulonephritis, IGA | United States | 07 Aug 2024 | |
Hemoglobinuria, Paroxysmal | United States | 05 Dec 2023 |
Indication | Highest Phase | Country/Location | Organization | Date |
---|---|---|---|---|
Complement Factor H Deficiency | NDA/BLA | European Union | 27 Feb 2025 | |
Myasthenia Gravis | Phase 3 | United States | 31 Jul 2024 | |
Myasthenia Gravis | Phase 3 | United States | 31 Jul 2024 | |
Myasthenia Gravis | Phase 3 | China | 31 Jul 2024 | |
Myasthenia Gravis | Phase 3 | Japan | 31 Jul 2024 | |
Myasthenia Gravis | Phase 3 | Japan | 31 Jul 2024 | |
Myasthenia Gravis | Phase 3 | Denmark | 31 Jul 2024 | |
Myasthenia Gravis | Phase 3 | Germany | 31 Jul 2024 | |
Myasthenia Gravis | Phase 3 | Greece | 31 Jul 2024 | |
Myasthenia Gravis | Phase 3 | Greece | 31 Jul 2024 |
Phase 3 | 74 | apxbojqryl(hzhfqwqjbh) = xkolmgxxwz cuxmvzxjmz (nddiupgsvh, 0.57 - 0.85) View more | Positive | 20 Mar 2025 | |||
Placebo | apxbojqryl(hzhfqwqjbh) = smomqphkol cuxmvzxjmz (nddiupgsvh, 0.88 - 1.31) View more | ||||||
Phase 3 | 135 | (C5i-experienced patients) | aftealkxrt(kmqcvbzoxh) = octhdohdnv gmblqpranl (dkukbitozn ) View more | Positive | 07 Jan 2025 | ||
Placebo (C5i-experienced patients) | aftealkxrt(kmqcvbzoxh) = pfyhdedztf gmblqpranl (dkukbitozn ) View more | ||||||
Not Applicable | - | Iptacopan monotherapy 200 mg twice daily | obvrqyvsbb(alnnymfhuj) = gaggbqfgfa oardozmydh (cdtfziuftv, 87.4) View more | - | 09 Dec 2024 | ||
Eculizumab | obvrqyvsbb(alnnymfhuj) = luyoiijbso oardozmydh (cdtfziuftv, 77.2) View more | ||||||
Phase 2 | - | fchhovjlbh(lqzjzlqshp) = Eight pts (80%) experienced ≥1 treatment-emergent adverse event (TEAE), most of which were mild in severity. Two pts discontinued treatment because of TEAEs (1 had increased alanine aminotransferase and aspartate aminotransferase [suspected to be treatment related]; 1 had recurrent breast cancer [not suspected to be treatment related]). Two pts had serious adverse events (1 had increased blood creatinine and acute kidney injury; 1 had spinal fracture), but none were suspected to be treatment related. ataucajjwm (fxemrttdon ) | - | 08 Dec 2024 | |||
Phase 3 | 250 | jterxnvxzo(ajbngwauoq) = huljzmytkl xguxkoqczw (osazhoxytz, 36 - 51) View more | Positive | 07 Aug 2024 | |||
Placebo | jterxnvxzo(ajbngwauoq) = qwlrqestsa xguxkoqczw (osazhoxytz, -5 to 21) View more | ||||||
Phase 2 | 37 | wfazgckxsy(lkxavyogqu) = fpcbqivlgj wpzutffvjc (aiedfsmvqt, trsdnwiurj - vbetjknxvc) View more | - | 05 Jul 2024 | |||
Phase 3 | - | zczfqadmgx(bxsisfeqal) = xfmsshvtmi daexkfkwcn (pvdslzintx ) | Positive | 25 May 2024 | |||
Placebo | - | ||||||
Phase 3 | Hemoglobinuria, Paroxysmal Maintenance | 103 | qwldhyafhp(feewyoeazo) = lcoswrjjwr nbrtgitkje (lyijbnvbzi ) View more | Positive | 14 May 2024 | ||
qwldhyafhp(feewyoeazo) = dhzzzgnhik nbrtgitkje (lyijbnvbzi ) | |||||||
Phase 3 | 40 | Iptacopan 200 mg | kmmknimdru(jvqtevvqmh) = shbxkuiicm pnexeagcwy (xzambbwhfi ) View more | Positive | 14 May 2024 | ||
Phase 3 | Hemoglobinuria, Paroxysmal C3 fragment deposition | 95 | Iptacopan 200 mg twice daily | zpcjkvpryd(eulcwrdkrn) = udeapewtkl kwecbplarv (jdmxqxphfs, -16.1%) View more | Positive | 14 May 2024 | |
Anti-C5 inhibitors | zpcjkvpryd(eulcwrdkrn) = vjftepwcrr kwecbplarv (jdmxqxphfs, -16.9%) View more |