Glycerol Phenylbutyrate

Summary

Glycerol Phenylbutyrate (CHEMBL2105745) is an approved small molecule (ATC A16AX09); indicated across 4 conditions including urea cycle disorder and parkinson disease.

At a glance

• Status: Approved (max clinical phase 4)
• Modality: Small molecule
• ATC class: A16AX09
• Indications: 4 conditions
• Clinical trials: 20
• Chemistry: 530.6 Da · C33H38O6

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL2105745
Name	Glycerol Phenylbutyrate
Type	Small molecule
Max phase	4
FDA approved	yes
PubChem CID	10482134
ATC	A16AX09
Molecular formula	C33H38O6
Molecular weight	530.6
InChIKey	ZSDBFLMJVAGKOU-UHFFFAOYSA-N

SMILES: C1=CC=C(C=C1)CCCC(=O)OCC(COC(=O)CCCC2=CC=CC=C2)OC(=O)CCCC3=CC=CC=C3

IUPAC name: 2,3-bis(4-phenylbutanoyloxy)propyl 4-phenylbutanoate

Also known as: Fenilbutirato de glicerol, Glycerol phenylbutyrate, HPN-100, HPN100, Phenylbutyrate de glycerol, Ravicti, GLYCEROL PHENYLBUTYRATE

Patent coverage: 58 distinct patent families (134 SureChEMBL compound mentions), from 1 matched compound structure(s). Mentions count patents naming the compound (not distinct inventions), so promiscuous / reference molecules inflate the mention figure — families are the dedup metric.

Targets

Targets

No target linkage available.

Bioactivity

No ChEMBL bioactivity rows at pChembl ≥ 5 (expected for biologics / antibodies).

Target pathways

No target-pathway data for this drug (no mapped target genes).

Indications & clinical

Indications

1 approved indication. FDA phase 4, plus an anticancer drug’s labelled cancer uses (which ChEMBL often logs at phase 3).

Indication	Phase	MONDO	EFO
urea cycle disorder	4	MONDO:0004739	MONDO:0004739

2 diseases in clinical trials (phase 1–3, investigational — not approved indications). Highest ChEMBL trial phase per disease; a non-cancer approved use is occasionally logged at phase 3 here.

Disease (in trials)	Phase	MONDO	EFO
Parkinson disease	1	MONDO:0005180	MONDO:0005180
cystic fibrosis	1	MONDO:0009061	MONDO:0009061

1 further indication record had no mapped disease name (EFO/MeSH-only) or were duplicates, and are omitted.

Clinical trials

Total trials: 20.

Phase distribution

Phase	Trials
PHASE2	5
PHASE1	5
PHASE4	3
PHASE3	3
Not specified	2
PHASE2/PHASE3	1
EARLY_PHASE1	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT01257737	PHASE4	COMPLETED	To Evaluate the Safety of Long-term Use of HPN-100 in the Management of Urea Cycle Disorders (UCDs)
NCT02246218	PHASE4	COMPLETED	A Study of the Safety, Efficacy and Pharmacokinetics of Glycerol Phenylbutyrate in Pediatric Subjects Under 2 Years of Age With Urea Cycle Disorders
NCT03335488	PHASE4	COMPLETED	Study of Glycerol Phenylbutyrate & Sodium Phenylbutyrate in Phenylbutyrate Naïve Patients With Urea Cycle Disorders (UCDs)
NCT00947297	PHASE3	COMPLETED	Study of the Safety of HPN (Hyperion)-100 for the Long-Term Treatment of Urea Cycle Disorders (Treat UCD)
NCT00992459	PHASE3	COMPLETED	Efficacy and Safety of HPN-100 for the Treatment of Adults With Urea Cycle Disorders
NCT01347073	PHASE3	COMPLETED	Study of the Safety, Pharmacokinetics and Efficacy of HPN-100, in Pediatric Subjects With Urea Cycle Disorders (UCDs)
NCT05019417	PHASE2/PHASE3	UNKNOWN	Glycerol-Phenylbutyrate Treatment in Children With MCT Mutation (Allan-Herndon- Dudley Syndrome)
NCT05983588	PHASE2	ACTIVE_NOT_RECRUITING	PROFIL Study to Investigate the Effect of GPB on NfL Levels in Patients With Corticobasal Syndrome (CBS)
NCT06887777	PHASE2	RECRUITING	Efficacy and Safety of the Treatment of Pyruvate Dehydrogenase Deficiency Patients With Glycerol Phenylbutyrate (RAVICTI)
NCT00551200	PHASE2	COMPLETED	Dose-Escalation Safety Study of HPN-100 to Treat Urea Cycle Disorders
NCT00947544	PHASE2	COMPLETED	Study of the Safety and Tolerability of HPN-100 Compared to Sodium Phenylbutyrate in Children With Urea Cycle Disorders
NCT00999167	PHASE2	COMPLETED	A Study of Safety and Efficacy of HPN-100 in Subjects With Cirrhosis and Episodic Hepatic Encephalopathy
NCT00977600	PHASE1	COMPLETED	A Study of Glyceryl Tri-(4-phenylbutyrate) (GT4P)
NCT00986895	PHASE1	COMPLETED	A Study of Glyceryl Tri-(4-phenylbutyrate) Administered Orally as a Single Dose, and Twice Daily for Seven Consecutive Days to Subjects With Hepatic Impairment With Cirrhosis and to a Control Group
NCT01135680	PHASE1	COMPLETED	Double-Blind Randomized Crossover Trial to Access Electrocardiogram Effects of HPN-100
NCT01881984	PHASE1	COMPLETED	Use of Ravicti™ in Patients With MCAD Deficiency With the 985A>G (K304E) Mutation
NCT02046434	PHASE1	COMPLETED	Phenylbutyrate Response as a Biomarker for Alpha-synuclein Clearance From the Brain
NCT04937062	EARLY_PHASE1	ACTIVE_NOT_RECRUITING	Phenylbutyrate for Monogenetic Developmental and Epileptic Encephalopathy
NCT01949766	Not specified	NO_LONGER_AVAILABLE	Transition From Buphenyl to RAVICTI for the Therapy of Byler Disease
NCT02094222	Not specified	NO_LONGER_AVAILABLE	Expanded Access Protocol for an Intermediate Size Population - RAVICTI for Byler Disease

Clinical evidence (CIViC)

No CIViC predictive evidence (expected for non-precision-medicine drugs).

Pharmacology

Pharmacogenomics

No CPIC/DPWG dosing guideline or drug-level clinical/variant annotations in PharmGKB for this molecule.

Related molecules

Related molecules

No competitor molecules sharing a primary target (ChEMBL phase ≥2 or PubChem drug-class).