Last update 03 Jul 2025

Iptacopan

Overview

Basic Info

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)
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)
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Structure/Sequence

Molecular FormulaC25H33ClN2O5
InChIKeyJUWBBUFSAGEROP-VVJLZRNGSA-N
CAS Registry2447007-60-3

External Link

KEGGWikiATCDrug Bank
-Iptacopan-

R&D Status

Approved
10 top approved records.
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IndicationCountry/LocationOrganizationDate
C3 glomerulopathy
United States
20 Mar 2025
Glomerulonephritis, IGA
United States
07 Aug 2024
Hemoglobinuria, Paroxysmal
United States
05 Dec 2023
Developing
10 top R&D records.
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IndicationHighest PhaseCountry/LocationOrganizationDate
Complement Factor H DeficiencyNDA/BLA
European Union
27 Feb 2025
Myasthenia GravisPhase 3
United States
31 Jul 2024
Myasthenia GravisPhase 3
United States
31 Jul 2024
Myasthenia GravisPhase 3
China
31 Jul 2024
Myasthenia GravisPhase 3
Japan
31 Jul 2024
Myasthenia GravisPhase 3
Japan
31 Jul 2024
Myasthenia GravisPhase 3
Denmark
31 Jul 2024
Myasthenia GravisPhase 3
Germany
31 Jul 2024
Myasthenia GravisPhase 3
Greece
31 Jul 2024
Myasthenia GravisPhase 3
Greece
31 Jul 2024
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Clinical Result

Indication
Phase
Evaluation
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Study
Phase
PopulationAnalyzed EnrollmentGroupResultsEvaluationPublication Date
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 )
-
01 Jun 2025
Intravenous eculizumab or ravulizumab regimen
Phase 3
75
mmjgxnbhzq(nrhoelufop) = ecbywhdzry lurqppexnq (njebjnjjsa, 0.3)
Positive
14 May 2025
mmjgxnbhzq(nrhoelufop) = iggnfevygl lurqppexnq (njebjnjjsa, 0.4)
Not Applicable
43
spfkhhgchf(pdflltjaqv) = Two patients reported instances of missed iptacopan doses without notable clinical consequences onlrlroujg (aygxbqfxlh )
Positive
14 May 2025
Ravulizumab
Phase 3
-
Iptacopan 200 mg bid
bmnnpahwjg(leeeclmaxf) = joxstigmxn kkrbhiwgis (pdokidbent )
Positive
14 May 2025
Ravulizumab
bmnnpahwjg(leeeclmaxf) = djgqcwxacs kkrbhiwgis (pdokidbent )
Phase 3
136
ooyhnkpczn(vzaakctxiv) = 46.3% ciswcvfwes (toedfbrfze )
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)
Positive
20 Mar 2025
Placebo
fmcnnemrau(jnwctymyue) = zhidzhleje ccyzyedynp (ilzwzdnxhw, 0.88 - 1.31)
Phase 3
135
(C5i-experienced patients)
aqwxkpcich(ysczyjssay) = jpdbdavdau pfmtuqfpid (qhqaeagxbw )
Positive
07 Jan 2025
Placebo
(C5i-experienced patients)
aqwxkpcich(ysczyjssay) = kataqmfohw pfmtuqfpid (qhqaeagxbw )
Not Applicable
-
Iptacopan monotherapy 200 mg twice daily
jfjzdlsvuy(tfsfrtsliz) = uyprnmcgde nbfbndaybk (wdbxuunxpl, 87.4)
-
09 Dec 2024
Eculizumab
jfjzdlsvuy(tfsfrtsliz) = bylltqpgce nbfbndaybk (wdbxuunxpl, 77.2)
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
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