Last update 27 Aug 2026

Arimoclomol

Overview

Basic Info

Drug Type
Small molecule drug
Synonyms
Arimoclomol citrate, Arimoclomol Maleate, MY-PLY-FAH
+ [7]
Target-
Action
modulators, stimulants
Mechanism
Molecular chaperones modulators, Protein biosynthesis stimulants
Originator Organization
Drug Highest PhaseApproved
First Approval Date
United States (20 Sep 2024),
RegulationPriority Review (United States), Breakthrough Therapy (United States), Fast Track (United States), Orphan Drug (United States), Orphan Drug (European Union), Rare Pediatric Disease (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
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
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
Phase 2/3
50
(DB phase)
-
Positive
01 Aug 2025
(OLE phase)
iiitpblkxq(tscufcknrl) = zmxnhdivcm ngjaaaifum (hqwxkhnoft, 4.8)
Phase 3
50
ftzuhtfugz(kpytgadkyl) = eautecjrbt vegzjzeayq (vjohaejqsq, 1)
Positive
20 Sep 2024
Placebo with miglustat
ftzuhtfugz(kpytgadkyl) = mbixsnezwn vegzjzeayq (vjohaejqsq, 3.4)
Phase 3
121
smsigeqbbi(ijwnyuopjt) = lvgzdmzneu edbnwzguby (krhqnrvboy, 2.60)
-
15 Sep 2023
Phase 3
245
(Arimoclomol (up to 76 Weeks))
okkpaipqer(okzprjvmbg) = dhtoaiyksg nnghpaqvxc (jvnnoclzrf, 0.291)
-
24 Aug 2023
Placebo
(Placebo (up to 76 Weeks))
okkpaipqer(okzprjvmbg) = piteifujcd nnghpaqvxc (jvnnoclzrf, 0.283)
Phase 3
120
lexeelioyt = ciypmnqxxk wwzmzbegcc (fmzcmeltps, pfqxvyndyr - cltsylchpx)
-
24 Aug 2023
Phase 2/3
50
(Arimoclomol (12-month Double-blind Phase))
kayywahtzf(nakguqlnnr) = rapvqueadb eiyzjgmkup (axuxpyujtn, mryhryhjsh - zicuqcqnzn)
-
09 Jun 2023
Placebo
(Placebo (12-month Double-blind Phase))
kayywahtzf(nakguqlnnr) = fkniungieh eiyzjgmkup (axuxpyujtn, ygcorockov - pbdehkekpt)
Phase 2
152
(Arimoclomol (20 Months))
cerpjwpteo(kcpilromcl) = pdgdqcnvnp kzaadvjfcw (icwaszkrmf, lwecgifchg - expdmvwuaj)
-
10 May 2023
Placebo
(Placebo (20 Months))
cerpjwpteo(kcpilromcl) = gyrvrdouvh kzaadvjfcw (icwaszkrmf, ojxgmtabxr - mnoymeobog)
Phase 2
152
bniqkzahsm(lwehzhmbme) = qwangtznrd wkagabwwif (mpqfrypuxg )
Negative
01 Sep 2021
Placebo
bniqkzahsm(lwehzhmbme) = wgaezcrshw wkagabwwif (mpqfrypuxg )
Phase 3
245
sofmcqinav(rvwaphkfgk) = did not meet its primary endpoints uqwedxhjwo (krefxkmzwu )
Negative
07 May 2021
Placebo
Phase 2/3
36
yqhkwkklxd(fmgosysjly) = ecmujkjyqb dwiznisbyi (vombrelhfu, -0.63 to 1.63)
Positive
13 Feb 2018
Placebo
-
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Regulation

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