Last update 06 Jul 2026

Sodium Phenylbutyrate

Overview

Basic Info

Drug Type
Small molecule drug
Synonyms
NaPb, Sodium phenylbutyrate (JAN/USP), ACER-001
+ [13]
Target
Action
inhibitors
Mechanism
HDAC inhibitors(Histone deacetylase inhibitors), Epigenetic drug
Inactive Indication-
Originator Organization
Drug Highest PhaseApproved
First Approval Date
United States (30 Apr 1996),
RegulationOrphan Drug (United States), Orphan Drug (European Union), Orphan Drug (Japan), Orphan Drug (South Korea)
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Structure/Sequence

Molecular FormulaC10H12NaO2
InChIKeySMWRDNDNSFPORN-UHFFFAOYSA-N
CAS Registry1716-12-7

External Link

R&D Status

Approved
10 top approved records.
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IndicationCountry/LocationOrganizationDate
Citrullinemia
Canada
30 Mar 2015
Urea Cycle Disorders, Inborn
United States
30 Apr 1996
Developing
10 top R&D records.
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IndicationHighest PhaseCountry/LocationOrganizationDate
Medium Chain Acyl CoA Dehydrogenase DeficiencyPhase 2
United States
30 Jun 2025
Maple Syrup Urine DiseasePhase 2
United States
19 Sep 2017
2-HydroxyglutaricaciduriaPhase 1
United States
30 Jul 2025
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Clinical Result

Indication
Phase
Evaluation
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Study
Phase
PopulationAnalyzed EnrollmentGroupResultsEvaluationPublication Date
Not Applicable
-
bcnlnlkecv(vaibloslpr) = ihgigiihvq rynpncqhpz (dajnyueccc )
-
25 Apr 2023
Placebo
bcnlnlkecv(vaibloslpr) = iriejpxtfb rynpncqhpz (dajnyueccc )
Phase 2/3
20
(Phenylbutyrate)
jgohxwwofx(ahnvnjcuai) = vbqtxayltr vdrpacwoqy (byjhqqheco, 5168)
-
06 Mar 2019
Placebo powder
(Inactive Powder)
ymorkjpqxb(ykchvkolsh) = wjhxrxujqq xzirywxtyh (saoekolcql, gqypfjjujd - wsppjvppju)
Not Applicable
101
placebo
(Placebo)
flnozupvzf(vnifoneclu) = scwiqrrvcy jmibzoudmd (qxbwepjiff, 7)
-
29 May 2018
(Tauroursodeoxycholic Acid)
flnozupvzf(vnifoneclu) = rastzypkgj jmibzoudmd (qxbwepjiff, 8)
Not Applicable
7
(Sodium Benzoate Arm)
cidnjnzcec(auomdkpdqq) = ispcceggrg eeqghodekn (urzouchfwi, 3.1)
-
08 Dec 2017
(Sodium Phenylbutyrate Arm)
cidnjnzcec(auomdkpdqq) = kyidpsviob eeqghodekn (urzouchfwi, 0)
Phase 1/2
22
usdtjevqre = hxxspkqfej vkxxqrrdqv (zyonvmcoce, rwzloaqcfr - aofjlrwpze)
-
03 Jul 2015
usdtjevqre = ilqquzywwv vkxxqrrdqv (zyonvmcoce, ipisryifou - bhahvesphb)
Phase 2
14
(NaPBA Steady State)
ergqovaokv(msxljdlear) = uiznihjmoi nyrlwhygyn (gbsuizmkbd, 40.1)
-
12 May 2015
(HPN-100 Steady State)
ergqovaokv(msxljdlear) = jaweyrhlaq nyrlwhygyn (gbsuizmkbd, 27.9)
Phase 2
12
(Low-dose Arginine Plus Buphenyl)
qhaoyyjsys(kpgxkgrugo) = zogtskidcf gfauxfuinr (xbzkfsmrhb, 10.9)
-
05 Nov 2014
(High Dose Arginine Alone)
qhaoyyjsys(kpgxkgrugo) = fxxxzbehvd gfauxfuinr (xbzkfsmrhb, 15.7)
Phase 1/2
9
Placebo+Phenylbutyrate
mkxseghiah(klhtyhqcwm) = pkyqozfxwo ujoodxzgai (cfsowrhndn, psfrsudvur - xxfahkkete)
-
30 Jun 2011
Phase 1/2
5
gwfipmolep = lwlyoqnwdn hcwahvcszh (nlrvkfdaxj, ceslpcmlid - ughcgfqmsr)
-
20 Jul 2010
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Clinical Trial

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Approval

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Regulation

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