Drugs / Glycerol phenylbutyrate
Glycerol phenylbutyrate
also known as
HPN-100 · Ravicti · GPB · Fenilbutirato de glicerol · Glyceryl Tri-(4-phenylbutyrate) · GT4P · PBA · Phenylbutyrate de glycerol · +1 more
Regulatory milestones approvals, filings & regulatory actions · 6 recorded
| Milestone | Jurisdiction | Brand | Indication | Date | Sentence it was read from |
|---|---|---|---|---|---|
| Label expansions (4) | US (FDA) | RAVICTI | — | 2016-09-08 – 2019-10-04 | fda.gov ↗ |
| Approved | EU (EMA) | Ravicti | — | 2015-11-26 | europa.eu ↗ |
| Approved | US (FDA) | RAVICTI | — | 2013-02-01 | fda.gov ↗ |
Trials 19 · a red edge is where a trial was stopped
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 02 trials | ||||||
| Phase 0 | NCT04041232 | Apr 2025 → Aug 2027 | achromatopsia 7 | Columbia University | Suspended | No outcome recorded |
| Phase 0 | NCT04937062 | Mar 2021 → Dec 2026 expected | genetic developmental and epileptic encephalopathy | Weill Medical College of Cornell University | Active not recruiting | No outcome recorded |
| Phase 14 trials | ||||||
| Phase 1 | NCT02046434 | Jan 2014 → Sep 2019 | Parkinson disease | University of Colorado, Denver | Completed | No outcome recorded |
| Phase 1 | NCT01881984 | Jun 2013 → Feb 2016 | medium chain acyl-CoA dehydrogenase deficiency | University of Pittsburgh | Completed | No outcome recorded |
| Phase 1 | NCT00986895 | Sep 2006 → Jun 2007 | hepatic encephalopathy, urea cycle disorder | Amgen | Completed | No outcome recorded |
| Phase 1 | NCT00977600 | Mar → Jul 2005 | — | Amgen | Completed | No outcome recorded |
| Phase 24 trials | ||||||
| Phase 2 | NCT06887777 | Oct 2025 → Apr 2026 overdue | pyruvate dehydrogenase deficiency | Assistance Publique - Hôpitaux de Paris | Recruiting | No outcome recorded |
| Phase 2 | NCT05983588 | Dec 2023 → Mar 2026 overdue | corticobasal syndrome | Technical University of Munich | Active not recruiting | No outcome recorded |
| Phase 2 | NCT00947544 | Mar 2010 → Aug 2011 | urea cycle disorder | Amgen | Completed | No outcome recorded |
| Phase 2 | NCT00999167 | Dec 2009 → Apr 2012 | cirrhosis of liver, hepatic encephalopathy | Amgen | Completed | No outcome recorded |
| Phase 2/31 trial | ||||||
| Phase 2/3 | NCT05019417 | Jun 2021 → Aug 2022 | disorder of metabolite absorption and transport | Kaplan Medical Center | Unknown | No outcome recorded |
| Phase 33 trials | ||||||
| Phase 3 | NCT01347073 | Jul 2011 → Feb 2013 | urea cycle disorder | Amgen | Completed | No outcome recorded |
| Phase 3 | NCT00947297 | Nov 2009 → Sep 2011 | urea cycle disorder | Amgen | Completed | No outcome recorded |
| Phase 3 | NCT00992459 | Oct 2009 → Sep 2010 | urea cycle disorder | Amgen | Completed | No outcome recorded |
| Phase 43 trials | ||||||
| Phase 4 | NCT03335488 | Feb 2018 → Jul 2022 | urea cycle disorder | Amgen | Completed | No outcome recorded |
| Phase 4 | NCT02246218 | Dec 2014 → Oct 2016 | urea cycle disorder | Amgen | Completed | No outcome recorded |
| Phase 4 | NCT01257737 | Oct 2010 → Feb 2017 | urea cycle disorder | Amgen | Completed | No outcome recorded |
| Phase not stated2 trials | ||||||
| — | NCT01949766 | — | MYO5B-related progressive familial intrahepatic cholestasis | University of Pittsburgh | No longer available | No outcome recorded |
| — | NCT02094222 | — | — | Robert Squires, Jr. | No longer available | No outcome recorded |
Also used as a comparator or background therapy in 3 trials
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 11 trial | ||||||
| Phase 1 | NCT01135680 Comparator | May → Sep 2010 | poisoning | Amgen | Completed | No outcome recorded |
| Phase 1/21 trial | ||||||
| Phase 1/2 | NCT02323100 Comparator | Dec 2018 → Mar 2022 | cystic fibrosis | National Jewish Health | Terminated | No outcome recorded |
| Phase 21 trial | ||||||
| Phase 2 | NCT00551200 Comparator | Oct 2007 → Jul 2008 | urea cycle disorder | Amgen | Completed | No outcome recorded |
Evidence & citations 12 cited values
Every value below carries the sentence it was read from. 26 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | Glycerol phenylbutyrate | “Glycerol phenylbutyrate (GPB), a liquid triglyceride pro-drug of PBA, containing no sodium and having favorable palatability, is being studied for treatment of hepatic...” PMID 20512995 ↗ Jun 20109“Glycerol phenylbutyrate (GPB), a prodrug of phenylbutyric acid, has demonstrated potential neuroprotective properties in preclinical studies on tauopathies.” PMID 42400090 ↗ Jul 2026 “Glycerol phenylbutyrate (PBA) is a triglyceride that consists of three molecules of phenylbutyrate linked to a glycerol backbone.” NCT04041232 ↗ “Phenylacetic acid (PAA) is the active moiety in sodium phenylbutyrate (NaPBA) and glycerol phenylbutyrate (GPB, HPN-100).” PMID 24144944 ↗ Oct 2013 “Glycerol phenylbutyrate” NCT05983588 ↗ |
| Known as | Fenilbutirato de glicerol | ChEMBL registry synonym — accepted as the source's own label CHEMBL2105745 ↗ |
| Known as | Glyceryl Tri-(4-phenylbutyrate) | “A Study of Glyceryl Tri-(4-phenylbutyrate) (GT4P)” NCT00977600 ↗ |
| Known as | GPB | “We hypothesize that treatment of AHDS patients with glycerol phenylbutyrate (GPB) will improve thyroid function and neurodevelopmental parameters and relieve symptoms resulting...” NCT05019417 ↗3“Glycerol phenylbutyrate (GPB), a liquid triglyceride pro-drug of PBA, containing no sodium and having favorable palatability, is being studied for treatment of hepatic...” PMID 20512995 ↗ Jun 2010 “Phenylacetic acid (PAA) is the active moiety in sodium phenylbutyrate (NaPBA) and glycerol phenylbutyrate (GPB, HPN-100).” PMID 24144944 ↗ Oct 2013 “RAVICTI 1.1 g/ml oral liquid (glycerol phenylbutyrate (GPB))” NCT05983588 ↗ |
| Known as | GT4P | “A Study of Glyceryl Tri-(4-phenylbutyrate) (GT4P)” NCT00977600 ↗ |
| Known as | HPN-100 | ClinicalTrials.gov intervention name — accepted as the source's own label NCT00992459 ↗ |
| Known as | PBA | “Glycerol phenylbutyrate (PBA) is a triglyceride that consists of three molecules of phenylbutyrate linked to a glycerol backbone.” NCT04041232 ↗ |
| Known as | Phenylbutyrate de glycerol | ChEMBL registry synonym — accepted as the source's own label CHEMBL2105745 ↗ |
| Known as | Ravicti | “Glycerol phenylbutyrate (trade name "Ravicti") is an FDA-approved medication used for urea cycle disorders in children and adults.” NCT04937062 ↗7“RAVICTI 1.1 g/ml oral liquid (glycerol phenylbutyrate (GPB))” NCT05983588 ↗ “Glycerol Phenylbutyrate 1100 MG/ML [Ravicti]” NCT06887777 ↗ |
| Known as | Ravicti, glycerol phenylbutyrate | “Ravicti, glycerol phenylbutyrate” NCT02323100 ↗ |
| Modality | Other / unclassified | “HPN-100 is a triglyceride that has a similar mechanism of action as NaPBA.” NCT00947544 ↗ |
| Route | Oral | “single oral dose of 9 mL HPN-100 given via syringes 3 times daily for 3 days” NCT01135680 ↗ |