Last update 13 Sep 2026

Glycerol Phenylbutyrate

Overview

Basic Info

Drug Type
Small molecule drug
Synonyms
Glycerol phenylbutyrate (USAN), Glyceryl Tri-4-Phenylbutyrate, GPB
+ [6]
Target-
Action
scavengers
Mechanism
Ammonia scavengers
Originator Organization
Inactive Organization
Drug Highest PhaseApproved
First Approval Date
United States (01 Feb 2013),
RegulationFast Track (United States), Orphan Drug (United States), Orphan Drug (European Union), Priority Review (China), Orphan Drug (Japan)
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Structure/Sequence

Molecular FormulaC33H38O6
InChIKeyZSDBFLMJVAGKOU-UHFFFAOYSA-N
CAS Registry611168-24-2

External Link

R&D Status

Approved
10 top approved records.
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IndicationCountry/LocationOrganizationDate
Urea Cycle Disorders, Inborn
United States
01 Feb 2013
Developing
10 top R&D records.
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IndicationHighest PhaseCountry/LocationOrganizationDate
Cystic FibrosisPhase 2
United States
-02 Dec 2018
FibrosisPhase 2
United States
01 Dec 2009
Hepatic EncephalopathyPhase 2
United States
01 Dec 2009
STXBP1-related epileptic encephalopathyPhase 1
United States
01 Mar 2021
Medium Chain Acyl CoA Dehydrogenase DeficiencyPhase 1
United States
-01 Jun 2013
Drug intoxicationPhase 1
United States
01 May 2010
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Clinical Result

Indication
Phase
Evaluation
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Study
Phase
PopulationAnalyzed EnrollmentGroupResultsEvaluationPublication Date
Phase 1/2
16
(Ravicti Low Dose)
aqqnslirpf(qvnxqttcwh) = szxntvhvyk zbfvikzuqi (wbmkkuxeaa, 3.54)
-
17 Apr 2026
Placebo
(Placebo)
aqqnslirpf(qvnxqttcwh) = hgucjkgzbv zbfvikzuqi (wbmkkuxeaa, 4.19)
Phase 4
88
HPN-100-012SE+HPN-100
(HPN-100- Pediatric)
bedqbjsaxh = kbpojcmrgg chtphnhldl (cskduwivgl, kooopdygmx - oqoamoynjz)
-
04 May 2018
HPN-100-012SE+HPN-100
(HPN-100 - Adult)
bedqbjsaxh = rkjaykubln chtphnhldl (cskduwivgl, liuktubngc - eudwakjhjn)
Phase 4
27
iyzccbuznn(jzwagdqzfu) = <100 vhnuhttihf (ekjpouhpav )
-
01 Nov 2017
Phase 1
4
qunaadmtzg(lfmzgwlvfk) = gogwzqwjkq ujbntvrzmd (bpqdbvsprk, cgzxsonawe - bhuuuwyqfu)
-
11 Jul 2017
Phase 2
189
(HPN-100)
wlquifjcok = atsjmqqvda bqtmmygkqb (xjdxooyrpp, wugkuqctwp - auwiehewwe)
-
30 Sep 2015
Placebo
(Placebo)
wlquifjcok = hseoqctzrk bqtmmygkqb (xjdxooyrpp, zlkmhfubym - auclmfudya)
Phase 2
14
(NaPBA Steady State)
jwgaroedom(niodmgkkdh) = eyjccdwcwa iybsuutirr (agpjqjhkhy, 40.1)
-
12 May 2015
(HPN-100 Steady State)
jwgaroedom(niodmgkkdh) = fzkxtvafzp iybsuutirr (agpjqjhkhy, 27.9)
Phase 2
178
tegeuejasm(vhplliahgt) = jjcdriofwe slhgkwipmi (nhykgganmb )
Positive
01 Mar 2014
Placebo
tegeuejasm(vhplliahgt) = qegmsqvoix slhgkwipmi (nhykgganmb )
Phase 3
60
mjqqbmuncr = jiorfrcuce zureqcllva (taudzmncoc, rbiwptlxzw - hcbmiwvjcv)
-
15 Aug 2013
Phase 3
46
(NaPBA)
pugmhpjymp(wjdjubxbqt) = peezwsvxwq enjhbpecpm (xbmitydhfk, 865.9)
-
12 Aug 2013
(HPN-100)
pugmhpjymp(wjdjubxbqt) = htrifgmcrr enjhbpecpm (xbmitydhfk, 660.53)
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Clinical Trial

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Approval

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Regulation

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