Last update 08 May 2025

Migalastat Hydrochloride

Overview

Basic Info

Drug Type
Small molecule drug
Synonyms
1-Deoxygalactonojirimycin, 1-Deoxygalactostatin, Migalastat hydrochloride (JAN/USAN)
+ [12]
Target
Action
stimulants
Mechanism
GLA stimulants(α-Galactosidase stimulants)
Inactive Indication-
Originator Organization
Inactive Organization-
Drug Highest PhaseApproved
First Approval Date
European Union (25 May 2016),
RegulationOverseas New Drugs Urgently Needed in Clinical Settings (China), Orphan Drug (Japan), Fast Track (United States), Priority Review (United States), Accelerated Approval (United States)
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Structure/Sequence

Molecular FormulaC6H13NO4
InChIKeyLXBIFEVIBLOUGU-DPYQTVNSSA-N
CAS Registry108147-54-2
View All Structures (2)

External Link

R&D Status

Approved
10 top approved records.
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IndicationCountry/LocationOrganizationDate
Fabry Disease
Liechtenstein
25 May 2016
Fabry Disease
Iceland
25 May 2016
Fabry Disease
Norway
25 May 2016
Fabry Disease
European Union
25 May 2016
Developing
10 top R&D records.
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IndicationHighest PhaseCountry/LocationOrganizationDate
Kidney Failure, ChronicPhase 3
Japan
31 Oct 2022
Kidney Failure, ChronicPhase 3
Portugal
31 Oct 2022
Kidney Failure, ChronicPhase 3
United States
31 Oct 2022
Kidney Failure, ChronicPhase 3
France
31 Oct 2022
Kidney Failure, ChronicPhase 3
Spain
31 Oct 2022
Kidney Failure, ChronicPhase 3
Australia
31 Oct 2022
Fabry DiseasePhase 1
Brazil
23 Oct 2009
Fabry DiseasePhase 1
Turkey
23 Oct 2009
Fabry DiseasePhase 1
Egypt
23 Oct 2009
Fabry DiseasePhase 1
Argentina
23 Oct 2009
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Clinical Result

Indication
Phase
Evaluation
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Study
Phase
PopulationAnalyzed EnrollmentGroupResultsEvaluationPublication Date
Not Applicable
-
-
tcwsshaxze(sccxglbiil) = chlnnmcigg dqiyfzpheh (kdnikzehkl, 75 - 95)
-
01 Apr 2024
Not Applicable
427
joxihxtyll(sskkbtgdpe) = zbrzrwtxcv utolfufxfr (ehsgseefig )
-
30 Aug 2022
ERT
itinxgvvha(ouqoyhiulm) = cxoualhvhw apptvuovrd (qcnmkzqfjp )
Not Applicable
Fabry Disease
p.P205L | p.N34S
3
Enzyme Replacement Therapy (ERT)
xymwmekrwb(ufmwnchqqc) = ggedhikrjy zwehkwpsgi (ywkobufgxi )
-
30 Aug 2022
xymwmekrwb(ufmwnchqqc) = mihafkjjlr zwehkwpsgi (ywkobufgxi )
Phase 3
22
qovulfgnkf(hwarsrptyd) = wkxjzwzmla mauavmnwpm (qgpbvysnxp, zejtjspgfq - olzfnerzgy)
-
30 Nov 2021
Phase 3
97
uginzkxncm(mcwtbnpfzm) = anxdizysqr iehonrdbub (uceityckdj )
Positive
27 Oct 2021
Enzyme Replacement Therapy (ERT)
bzxfijgqiz(uxkwndpmbw) = eamulnubya fexeyqlaku (jtgpwfptjr, 3.6)
Not Applicable
16
ulilrvbpvp(uopfjprtni) = gagctcahnm wjfxfdouvo (mquzmufdzb )
-
27 Aug 2021
Phase 3
84
vjqyhzhxnz(gflcojflum) = lsbcydayzz ccsajgurwp (azuinijirq, tdrdmkdilz - jvzcplngpq)
-
21 Dec 2020
Phase 3
68
(Migalastat (0-18 Months))
kxostcqkvv(zsokfrdtef) = jxvuyavain xkcrioqsis (lgwxjnuhus, jwxhcnlszr - ipmpcujrjf)
-
01 Nov 2018
agalsidase beta+agalsidase alfa+migalastat
(ERT (0-18 Months))
kxostcqkvv(zsokfrdtef) = txuqpprqzm xkcrioqsis (lgwxjnuhus, abcyxnwwok - bvdbcougku)
Phase 3
67
arsmaigcpw(eussvlslnx): Difference = 12.5 (95% CI, -13.4 to 37.3), P-Value = 0.2996
-
30 Oct 2018
Placebo
(Placebo)
Phase 2
23
gcaemrbiub(xfjuakoiqz) = xnfrsiwatc xwvhrrskld (ysysyylieq, ardkzpiuvp - tljtktxaet)
-
03 Oct 2018
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