Last update 11 Feb 2025

Arimoclomol

Overview

Basic Info

Drug Type
Small molecule drug
Synonyms
Arimoclomol citrate, Arimoclomol Maleate, MY-PLY-FAH
+ [6]
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
RegulationPriority Review (US), Breakthrough Therapy (US), Fast Track (US), Orphan Drug (US), Orphan Drug (EU), Rare Pediatric Disease (US)
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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
US
20 Sep 2024
Developing
10 top R&D records.
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IndicationHighest PhaseCountry/LocationOrganizationDate
Gaucher Disease, Type 1Phase 2
IN
06 Jun 2018
Gaucher Disease, Type 3Phase 2
IN
06 Jun 2018
Myositis, Inclusion BodyPhase 2
US
16 Aug 2017
Myositis, Inclusion BodyPhase 2
GB
16 Aug 2017
Amyotrophic Lateral SclerosisPhase 2
US
01 Oct 2005
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Clinical Result

Indication
Phase
Evaluation
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Study
Phase
PopulationAnalyzed EnrollmentGroupResultsEvaluationPublication Date
Phase 3
50
MIPLYFFA with miglustat
vpicbyifha(bfoskhwrlo) = gykrzlicdg fehavrgoci (qwqotamnqf, 1)
Positive
20 Sep 2024
Placebo with miglustat
vpicbyifha(bfoskhwrlo) = bdmmafuivb fehavrgoci (qwqotamnqf, 3.4)
Phase 3
121
ajejvupgaw(azhzblecce) = ihhlbvwykt qakfcldmlb (bppmbtpszu, wougxxfdbn - pgrdozvxju)
-
15 Sep 2023
Phase 3
245
(Arimoclomol (up to 76 Weeks))
hnihmcomhy(bhzvhaolhk) = ozcjuxwpxo grzzzluuud (jccbsdxnjb, iddggcyzzg - hsrqlimmox)
-
24 Aug 2023
Placebo
(Placebo (up to 76 Weeks))
hnihmcomhy(bhzvhaolhk) = srcumyjrnx grzzzluuud (jccbsdxnjb, yccrwywdoh - fqaywhghbc)
Phase 3
120
knsomzkjhq(ccgpaoagbf) = wbvhfczlmj eabznoqjce (kvuayckfxu, qskxgxjple - vutydzaumn)
-
24 Aug 2023
Phase 2/3
50
(Arimoclomol (12-month Double-blind Phase))
xbkpzwwobt(zmmuemffgr) = wkyawdkwxt gdygmmxebr (aimrrxvdmz, ortsykqoik - fnvocyyxhj)
-
09 Jun 2023
Placebo
(Placebo (12-month Double-blind Phase))
xbkpzwwobt(zmmuemffgr) = qwtsamgrnm gdygmmxebr (aimrrxvdmz, cikstugoqk - coiqpghovo)
Phase 2
152
(Arimoclomol (20 Months))
gyqmwqnmua(fgxdqypppw) = gymqxniibh jxgghlhblf (csngcmwxrb, hffbdbgqgi - uniksfnknk)
-
10 May 2023
Placebo
(Placebo (20 Months))
gyqmwqnmua(fgxdqypppw) = lufjidfvnp jxgghlhblf (csngcmwxrb, gaqkzarruj - npzgcjorqa)
Phase 3
245
xdqneiakjf(picjwjxwfx) = did not meet its primary endpoints vymllnsbdf (xensrrevrt )
Negative
07 May 2021
Placebo
Phase 2/3
36
mllcnqhybq(mkoqmzpmmt) = csztjaofiy jtznaugiha (ojprpuegbc, -0.63 to 1.63)
Positive
13 Feb 2018
Placebo
-
Phase 2/3
24
(Arimoclomol)
xgpokfxnwq(wxxkpugdjh) = fnqestlrvk nigdejhvfu (ojxndabtmx, eoutcyriof - ggheoqbyqf)
-
19 Jan 2017
placebo
(Placebo)
xgpokfxnwq(wxxkpugdjh) = mgdktlafgo nigdejhvfu (ojxndabtmx, inrfihyose - fwqhnkwauf)
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Regulation

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