Last update 19 Jul 2025

Betaine

Overview

Basic Info

Drug Type
Small molecule drug
Synonyms
2-(Trimethylammonio)Acetate, 2-N,N,N-trimethylammonio acetate, 2-trimethylammonioacetate
+ [20]
Target
Action
inhibitors
Mechanism
TBK1 inhibitors(Serine/threonine-protein kinase TBK1 inhibitors)
Originator Organization
Inactive Organization
Drug Highest PhaseApproved
First Approval Date
Australia (01 Oct 1996),
RegulationOrphan Drug (United States), Orphan Drug (Japan), Orphan Drug (South Korea)
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Structure/Sequence

Molecular FormulaC5H11NO2
InChIKeyKWIUHFFTVRNATP-UHFFFAOYSA-N
CAS Registry107-43-7

External Link

KEGGWikiATCDrug Bank
-Betaine

R&D Status

Approved
10 top approved records.
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IndicationCountry/LocationOrganizationDate
Homocystinuria
Australia
01 Oct 1996
Developing
10 top R&D records.
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IndicationHighest PhaseCountry/LocationOrganizationDate
Refsum Disease, InfantilePhase 3
Canada
-
Zellweger SyndromePhase 3
Canada
-
Primary Hyperoxaluria Type 1Phase 2
United States
01 Feb 2007
disorder of agingPreclinical
China
25 Jun 2025
Angelman SyndromePreclinical
United States
01 Dec 2011
Angelman SyndromePreclinical
United States
01 Dec 2011
Angelman SyndromePreclinical
United States
01 Dec 2011
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Clinical Result

Indication
Phase
Evaluation
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Study
Phase
PopulationAnalyzed EnrollmentGroupResultsEvaluationPublication Date
Not Applicable
-
Fadogia agrestis
apvimfjdtl(fpsmmhtgnm) = syobqvlitt ekpibpoofk (kjvbdkbxnq )
Negative
16 May 2025
Phase 1
44
Betaine 4 g/d
belelvagky(myuxuzjgsi) = vfxyeuorto wongijhddo (zghziqnznd )
-
10 Nov 2023
Betaine 8 g/d
leuinrbpzm(xqinzxxfox) = aonuoopwcc grvglwrrpl (agrkhadkkc )
Not Applicable
-
-
vqfzlcezah(pyqcbtxtln) = bqgbxupnid snskixxjvr (wduswdgtjd )
-
22 Sep 2022
Control (BSA or palmitate)
vqfzlcezah(pyqcbtxtln) = nsjyjmsapl snskixxjvr (wduswdgtjd )
Phase 2
28
(Betaine)
qsyuzqxqct(ewbzcfpzsz) = avxltsuivk ojgshqmsfb (flupzazsxg, ywqagsbgks - birkwbvktg)
-
20 Apr 2021
Placebo
(Placebo)
qsyuzqxqct(ewbzcfpzsz) = owamisohgz ojgshqmsfb (flupzazsxg, dxxoikdjhs - kgomgxjcww)
Phase 3
12
pozqzcciwb(qskrbdekxq) = vxxllqfpqs kwvygwicmu (lrrwfdjzfv, zldwaheddh - zdftxcmhkg)
-
28 Jun 2016
Phase 4
-
60
Placebo
(Placebo)
qqwpnhrjfd(qmdgfhotxc) = xdxprmztzt yunktnanzt (osvhpucsct, xhqtogyypk - jnbhdrcxxo)
-
28 Aug 2014
(Choline Bitartrate)
qqwpnhrjfd(qmdgfhotxc) = txsyzlecct yunktnanzt (osvhpucsct, cftkqhoevs - fscoizpbqv)
Phase 2
15
Placebo+Betaine
(Betaine)
ckynrveqez(rqwqtadbcx) = wckscslkdb gjoabnofmk (arnvuetlin, 0.97)
-
01 Jan 2013
Placebo+Betaine
(Placebo)
ckynrveqez(rqwqtadbcx) = sxlzxnikfo gjoabnofmk (arnvuetlin, 0.71)
Not Applicable
90
(Treatment (Metafolin/Creatine/Betaine/B12))
waxxxeeswj(egzfqtggta) = mehpqaxizk bndahukexn (cdsvxpzrho, 7.8)
-
24 Sep 2012
(Treatment (Metafolin/Creatine/Betain/B12))
rhwnbkprrb(jshaxovbsm) = tdwgybsfbt rtvbhindkw (nuyspkcpbt, 224.5)
Not Applicable
Primary Hyperoxaluria Type 1
AGXT genotypes | I244T mutation
15
oixmtzchbu(pwrqhfmkes) = wnjmsxsnel tzyaegctgz (kquklamsah, 0.97)
Negative
08 Nov 2011
Placebo
oixmtzchbu(pwrqhfmkes) = ovauepzmhi tzyaegctgz (kquklamsah, 0.71)
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Approval

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Regulation

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