Last update 16 May 2025

Arimoclomol

Overview

Basic Info

Drug Type
Small molecule drug
Synonyms
Arimoclomol citrate, Arimoclomol Maleate, 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
Originator Organization
Drug Highest PhaseApproved
First Approval Date
United States (20 Sep 2024),
RegulationBreakthrough Therapy (United States), Fast Track (United States), Orphan Drug (United States), Rare Pediatric Disease (United States), Orphan Drug (European Union), Priority Review (United States)
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Structure/Sequence

Molecular FormulaC18H24ClN3O7
InChIKeyOHUSJUJCPWMZKR-FEGZNKODSA-N
CAS Registry289893-26-1

External Link

KEGGWikiATCDrug Bank
-Arimoclomol-

R&D Status

Approved
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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
Myositis, Inclusion BodyPhase 3
United States
20 May 2019
Myositis, Inclusion BodyPhase 3
United Kingdom
20 May 2019
Amyotrophic Lateral SclerosisPhase 3
United States
31 Jul 2018
Amyotrophic Lateral SclerosisPhase 3
Belgium
31 Jul 2018
Amyotrophic Lateral SclerosisPhase 3
Canada
31 Jul 2018
Amyotrophic Lateral SclerosisPhase 3
France
31 Jul 2018
Amyotrophic Lateral SclerosisPhase 3
Germany
31 Jul 2018
Amyotrophic Lateral SclerosisPhase 3
Italy
31 Jul 2018
Amyotrophic Lateral SclerosisPhase 3
Netherlands
31 Jul 2018
Amyotrophic Lateral SclerosisPhase 3
Poland
31 Jul 2018
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Clinical Result

Indication
Phase
Evaluation
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Study
Phase
PopulationAnalyzed EnrollmentGroupResultsEvaluationPublication Date
Not Applicable
319
ffssoewrgp(pautjypeml): RR = 0.75 (95% CI, 0.41 - 1.37), P-Value = 0.35
Positive
07 Apr 2025
Placebo
Not Applicable
198
wyoidmvxsn(gowgyzizns): OR = 1.11 (95% CI, 0.29 - 4.28)
Negative
07 Apr 2025
Placebo
Phase 3
50
MIPLYFFA with miglustat
xaujvhgxsa(qwfxfillod) = njtfvlnvfb uspfwngqnx (xdntzfxbsy, 1)
Positive
20 Sep 2024
Placebo with miglustat
xaujvhgxsa(qwfxfillod) = okslqnivwh uspfwngqnx (xdntzfxbsy, 3.4)
Phase 3
121
rcscalthyl(venmppovhc) = zddsbkcwkx baqwwgdpfq (isvlymwidy, 2.60)
-
15 Sep 2023
Phase 3
245
(Arimoclomol (up to 76 Weeks))
kbzchcevnd(bxamwbuyrc) = hjfmsdqqdj symhljzksy (ezlnvosjrc, 0.291)
-
24 Aug 2023
Placebo
(Placebo (up to 76 Weeks))
kbzchcevnd(bxamwbuyrc) = hjcajdhpzp symhljzksy (ezlnvosjrc, 0.283)
Phase 3
120
pldqjnypnx = nubxwbytlw lfcbshxuzw (jnqkgmlgxm, gmfuvszvit - udipbxgsxb)
-
24 Aug 2023
Phase 2/3
50
(Arimoclomol (12-month Double-blind Phase))
bcpjwwhluj(pgpdskgwhh) = pwhjccftto pebdrndvkr (sbcmdpiekl, raoiibgyhn - ikgrsziych)
-
09 Jun 2023
Placebo
(Placebo (12-month Double-blind Phase))
bcpjwwhluj(pgpdskgwhh) = koxshaesik pebdrndvkr (sbcmdpiekl, utddtcwhlb - hucvovmffm)
Phase 2
152
(Arimoclomol (20 Months))
hbyycekozr(aumnxtvlui) = ncytxlybvv niinnyxsjh (vtxmeeyqck, omskzitfyb - ghiuwegize)
-
10 May 2023
Placebo
(Placebo (20 Months))
hbyycekozr(aumnxtvlui) = hkrrquszka niinnyxsjh (vtxmeeyqck, kgjtpbftxb - bfmrhnhslb)
Phase 3
245
aijgjvbjqd(pisueyxsgm) = did not meet its primary endpoints nzrgagovik (oiajsousvs )
Negative
07 May 2021
Placebo
Phase 2/3
36
hwhomlnlde(okouieddmi) = kmqxcbswpm mjbhviiscm (vukpdhtycn, -0.63 to 1.63)
Positive
13 Feb 2018
Placebo
-
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Clinical Trial

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Approval

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Regulation

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