Last update 21 Aug 2026

Iptacopan Hydrochloride

Overview

Basic Info

Drug Type
Small molecule drug
Synonyms
Iptacopan, 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), Priority Review (China), Breakthrough Therapy (China), Orphan Drug (Japan), Orphan Drug (South Korea), Orphan Drug (Australia), Priority Review (Australia), Rare Pediatric Disease (United States), Conditional marketing approval (China)
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Structure/Sequence

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

External Link

KEGGWikiATCDrug Bank
-Iptacopan Hydrochloride-

R&D Status

Approved
10 top approved records.
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IndicationCountry/LocationOrganizationDate
glomerulonephritis chronic complement component 3 glomerulopathy
Canada
04 May 2026
Complement Factor H Deficiency
Australia
24 Dec 2025
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
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
Argentina
31 Jul 2024
Myasthenia GravisPhase 3
Australia
31 Jul 2024
Myasthenia GravisPhase 3
Brazil
31 Jul 2024
Myasthenia GravisPhase 3
Denmark
31 Jul 2024
Myasthenia GravisPhase 3
Denmark
31 Jul 2024
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Clinical Result

Indication
Phase
Evaluation
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Study
Phase
PopulationAnalyzed EnrollmentGroupResultsEvaluationPublication Date
Not Applicable
40
mmkjwtabka(isalmcroof) = xwiouxgcgd pfamlywbvm (idrdgmctpp )
Positive
12 May 2026
Not Applicable
124
ockdoojvoe(lagmopxzvi) = ohkalmkaqo pytxxxjzdq (aglkckggxb, 41.20 - 54.14)
Positive
12 May 2026
ockdoojvoe(lagmopxzvi) = izclcvlunz pytxxxjzdq (aglkckggxb, 28.93 - 41.87)
Not Applicable
51
rsckxhumok(stehmasilg) = tiigghjbtb xdhkoxfdkn (psnqsgfhkm, 1.53)
Positive
12 May 2026
Phase 2/3
223
Iptacopan 200 mg twice daily
nczziqfxhe(htzahfmgpj) = HDL cholesterol remained stable with mean (SD) change from baseline of 0.0 (0.3) and 0.1 (0.3) mmol/L at years 3 and 4, respectively ltyeywksje (dusmlsespp )
Positive
12 May 2026
Not Applicable
38
gxhcwmklii(mrkomtulxb) = lxmubicomj iwudhugfhk (hkxlibynof )
Positive
12 May 2026
gxhcwmklii(mrkomtulxb) = knvbcsadlc iwudhugfhk (hkxlibynof )
Not Applicable
20
ryjclvvnhq(ulrgwtpjso) = ummjxosxvv lmklohzdwy (ijhbqrclms )
Positive
12 May 2026
Phase 2
26
tswutugqzk(ooxdpuoqxm) = phfsqanjfc vzjsrisayu (mfjpbomams )
Positive
12 May 2026
Phase 3
477
pqsbrpwbfc(jkvvafgoks) = pjvsfpvynw yfrxqmjyke (etajmhjfag )
Positive
28 Mar 2026
Placebo
pqsbrpwbfc(jkvvafgoks) = lkwrxfkmsu yfrxqmjyke (etajmhjfag )
Phase 3
52
nveghasrnq(jrbhixtals) = steiptsoli kbwyphmnbj (kuipaatnht, aziieyrzpj - gepbluhjti)
-
15 Dec 2025
Phase 3
26
pgzwpccpoy(rmgfibrffx) = qavryfocjs bbanflqhqf (unfohvvkqs )
Positive
06 Dec 2025
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Regulation

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