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 |
Not Applicable | - | Iptacopan 200 mg twice daily | yamaitsvdd(wonwygbebx) = Clinical breakthrough haemolysis occurred in seven (7%) of 96 iptacopan-treated patients in APPLY-PNH (including both groups) and two (5%) of 40 in APPOINT-PNH, but it was generally mild or moderate with no iptacopan discontinuation nebbtdwnxh (wytlqhampm ) View more | - | 01 Jun 2025 | ||
Intravenous eculizumab or ravulizumab regimen | |||||||
Phase 3 | 75 | mmjgxnbhzq(nrhoelufop) = ecbywhdzry lurqppexnq (njebjnjjsa, 0.3) View more | Positive | 14 May 2025 | |||
mmjgxnbhzq(nrhoelufop) = iggnfevygl lurqppexnq (njebjnjjsa, 0.4) View more | |||||||
Not Applicable | 43 | spfkhhgchf(pdflltjaqv) = Two patients reported instances of missed iptacopan doses without notable clinical consequences onlrlroujg (aygxbqfxlh ) View more | Positive | 14 May 2025 | |||
Ravulizumab | |||||||
Phase 3 | - | Iptacopan 200 mg bid | bmnnpahwjg(leeeclmaxf) = joxstigmxn kkrbhiwgis (pdokidbent ) View more | Positive | 14 May 2025 | ||
Ravulizumab | bmnnpahwjg(leeeclmaxf) = djgqcwxacs kkrbhiwgis (pdokidbent ) View more | ||||||
Phase 3 | 136 | ooyhnkpczn(vzaakctxiv) = 46.3% ciswcvfwes (toedfbrfze ) View more | Positive | 14 May 2025 | |||
Not Applicable | - | 30 | uahrdezoyd(svvcznbedi) = One bacterial infection was reported; a cystitis (Klebsiella) which did not lead to any iptacopan modification or discontinuation waljfxpzjq (hxwqrwcnib ) | Positive | 14 May 2025 | ||
(Compassionate use) | |||||||
Phase 3 | 74 | fmcnnemrau(jnwctymyue) = mzaziitvsf ccyzyedynp (ilzwzdnxhw, 0.57 - 0.85) View more | Positive | 20 Mar 2025 | |||
Placebo | fmcnnemrau(jnwctymyue) = zhidzhleje ccyzyedynp (ilzwzdnxhw, 0.88 - 1.31) View more | ||||||
Phase 3 | 135 | (C5i-experienced patients) | aqwxkpcich(ysczyjssay) = jpdbdavdau pfmtuqfpid (qhqaeagxbw ) View more | Positive | 07 Jan 2025 | ||
Placebo (C5i-experienced patients) | aqwxkpcich(ysczyjssay) = kataqmfohw pfmtuqfpid (qhqaeagxbw ) View more | ||||||
Not Applicable | - | Iptacopan monotherapy 200 mg twice daily | jfjzdlsvuy(tfsfrtsliz) = uyprnmcgde nbfbndaybk (wdbxuunxpl, 87.4) View more | - | 09 Dec 2024 | ||
Eculizumab | jfjzdlsvuy(tfsfrtsliz) = bylltqpgce nbfbndaybk (wdbxuunxpl, 77.2) View more | ||||||
Phase 2 | - | twmwrdvvvl(isiypwrkpw) = 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. yccppotbqj (gcbttksbmq ) | - | 08 Dec 2024 |