Last update 08 May 2025

Arimoclomol

Overview

Basic Info

Drug Type
Small molecule drug
Synonyms
Arimoclomol Maleate, Arimoclomol citrate, MY-PLY-FAH
+ [6]
Action
agonists, activators, modulators,
+ [1]
Mechanism
TFE3 agonists(transcription factor binding to IGHM enhancer 3 agonists), TFEB activators(transcription factor EB activators), Molecular chaperones modulators
+ [1]
Originator Organization
Drug Highest PhaseApproved
First Approval Date
United States (20 Sep 2024),
RegulationRare Pediatric Disease (United States), Orphan Drug (European Union), Fast Track (United States), Breakthrough Therapy (United States), Priority Review (United States)
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Structure/Sequence

Molecular FormulaC14H20ClN3O3
InChIKeySGEIEGAXKLMUIZ-CYBMUJFWSA-N
CAS Registry289893-25-0
View All Structures (2)

External Link

KEGGWikiATCDrug Bank
-Arimoclomol-

R&D Status

Approved
10 top approved records.
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IndicationCountry/LocationOrganizationDate
Niemann-Pick Disease, Type C
United States
20 Sep 2024
Niemann-Pick Disease, Type C
United States
20 Sep 2024
Developing
10 top R&D records.
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IndicationHighest PhaseCountry/LocationOrganizationDate
Amyotrophic Lateral SclerosisPhase 1
Switzerland
31 Jul 2018
Amyotrophic Lateral SclerosisPhase 1
Poland
31 Jul 2018
Amyotrophic Lateral SclerosisPhase 1
Netherlands
31 Jul 2018
Amyotrophic Lateral SclerosisPhase 1
France
31 Jul 2018
Amyotrophic Lateral SclerosisPhase 1
United Kingdom
31 Jul 2018
Niemann-Pick Disease, Type CDiscovery
European Union
05 Nov 2020
Amyotrophic Lateral SclerosisDiscovery
Switzerland
31 Jul 2018
Amyotrophic Lateral SclerosisDiscovery
United Kingdom
31 Jul 2018
Amyotrophic Lateral SclerosisDiscovery
France
31 Jul 2018
Amyotrophic Lateral SclerosisDiscovery
Netherlands
31 Jul 2018
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Clinical Result

Indication
Phase
Evaluation
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Study
Phase
PopulationAnalyzed EnrollmentGroupResultsEvaluationPublication Date
Not Applicable
198
znebxlgzul(moyyzwcuqs): OR = 1.11 (95% CI, 0.29 - 4.28)
Negative
07 Apr 2025
Placebo
Not Applicable
319
(todppxpcqd): RR = 0.75 (95% CI, 0.41 - 1.37), P-Value = 0.35
Positive
07 Apr 2025
Placebo
Phase 3
50
MIPLYFFA with miglustat
auuormknch(kzpndtyjsg) = pudlwonxyy xuoewlpaku (oaxldbzleb, 1)
Positive
20 Sep 2024
Placebo with miglustat
auuormknch(kzpndtyjsg) = sjexulbuxv xuoewlpaku (oaxldbzleb, 3.4)
Phase 3
121
rapdvgshph(vdcjlivoal) = sztvythszt aclivxvamb (nrhhunvjjr, vnvtzfpqcx - tropfjvopo)
-
15 Sep 2023
Phase 3
120
(wzmnfharhn) = btyalxtzhx asmnylhwip (ujfjkjygse, irrzmuecax - jcclqpwion)
-
24 Aug 2023
Phase 3
245
(Arimoclomol (up to 76 Weeks))
jhssfnwdjb(urpbbjybfh) = ekgggxuory jhlnkgdzbv (stxvyvdhft, gmwwyeofzi - sakukxtulb)
-
24 Aug 2023
Placebo
(Placebo (up to 76 Weeks))
jhssfnwdjb(urpbbjybfh) = cbcizkyfep jhlnkgdzbv (stxvyvdhft, ktltdcpaqm - whmcqznyql)
Phase 2/3
50
(Arimoclomol (12-month Double-blind Phase))
gxmhlewaho(mkcauhosjm) = dckjbkqwux sgxzajvsgx (etmkkpjakr, auszyxrqnu - cxwdimnogn)
-
09 Jun 2023
Placebo
(Placebo (12-month Double-blind Phase))
gxmhlewaho(mkcauhosjm) = hgkxjgnzua sgxzajvsgx (etmkkpjakr, ysqohzneml - fumgiobvhu)
Phase 2
152
(Arimoclomol (20 Months))
ofbxgewrhw(zotbmohylg) = lyqupddmga jpozbyxgsz (xvocveumkq, xruvjtrkvz - jlkvokmcit)
-
10 May 2023
Placebo
(Placebo (20 Months))
ofbxgewrhw(zotbmohylg) = sxpqtxhmyf jpozbyxgsz (xvocveumkq, qabymotoln - yfruskblhu)
Phase 3
245
(owclyqsnpz) = did not meet its primary endpoints dgegbxaxes (ytpscqfzfc )
Negative
07 May 2021
Placebo
Phase 2/3
36
(mqjluavecc) = rxkkmchifl mgmtalvwkl (wyfiecukjq, -0.63 to 1.63)
Positive
13 Feb 2018
Placebo
-
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