Drugs / Glycerol phenylbutyrate
Glycerol phenylbutyrate
also known as
HPN-100 · Ravicti · GPB · 4-PBA · 4-phenylbutyrate · Fenilbutirato de glicerol · Glyceryl Tri-(4-phenylbutyrate) · GT4P · +3 more
Regulatory milestones approvals, filings & regulatory actions · 9 recorded
| Milestone | Jurisdiction | Brand | Indication | Date | Sentence it was read from |
|---|---|---|---|---|---|
| Filed urea cycle disorder 3 milestones | |||||
| Filed | Japan (PMDA) | Ravicti | urea cycle disorder | 2024-12-27 | “OrphanPacific, Inc has submitted an application to the Ministry of Health, Labour and Welfare in Japan for the approval of Ravicti® (glycerol phenylbutyrate)” immedica.com ↗ |
| Filed | China (NMPA) | RAVICTI | urea cycle disorder | 2022-08-22 | “announced today that the National Medical Products Administration (NMPA) has accepted and granted Priority Review for the New Drug Application (NDA) of “glycerol phenylbutyrate”” immedica.com ↗ “RAVICTI® (glycerol phenylbutyrate), has been accepted and granted Priority Review for the New Drug Application (NDA) in China.” immedica.com ↗ |
| Filed | Other | RAVICTI | urea cycle disorder | — | “In April, another submission for RAVICTI® Market Authorisation Approval was accepted by the Department of Health (DoH) in HongKong.” immedica.com ↗ “In April, another submission for RAVICTI Market Authorisation Approval was accepted by the Department of Health (DoH) in HongKong.” immedica.com ↗ |
| Approved Indication not stated 3 milestones | |||||
| 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 20
| 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 25 trials · 1 met primary | ||||||
| Phase 2 | NCT07847918 | Oct 2026 → Mar 2029 expected | inborn disorder of neurotransmitter metabolism and transport | Scripps Translational Science Institute | Recruiting | No outcome recorded |
| 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 → Aug 2026 | corticobasal syndrome | Technical University of Munich | Completed | 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 | Met primary |
| 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 · 1 met primary | ||||||
| Phase 3 | NCT01347073 | Jul 2011 → Feb 2013 | urea cycle disorder | Amgen | Completed | Met primary |
| 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 · 1 missed | ||||||
| Phase 4 | NCT03335488 | Feb 2018 → Jul 2022 | urea cycle disorder | Amgen | Completed | Missed primary |
| 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 | ||||||
| — | NCT02094222 | — | — | Robert Squires, Jr. | No longer available | No outcome recorded |
| — | NCT01949766 | — | MYO5B-related progressive familial intrahepatic cholestasis | University of Pittsburgh | No longer available | No outcome recorded |
| Comparator or background therapy3 trials | ||||||
| Phase 1 | NCT01135680 Comparator | May → Sep 2010 | poisoning | Amgen | Completed | No outcome recorded |
| Phase 1/2 | NCT02323100 Comparator | Dec 2018 → Mar 2022 | cystic fibrosis | National Jewish Health | Terminated | No outcome recorded Stop: Business |
| Phase 2 | NCT00551200 Comparator | Oct 2007 → Jul 2008 | urea cycle disorder | Amgen | Completed | No outcome recorded |
News releases announcing trial results or a regulatory action · 11
| Date | Issuer | Release |
|---|---|---|
| 2026-05-05 | Lupin Limited | Regulatory Lupin Receives U.S. FDA Approval for Glycerol Phenylbutyrate Oral Liquid lupin.com ↗
Lupin Limited (Lupin) today announced that it has received approval from the United States Food and Drug Administration for its Abbreviated New Drug Applications for Glycerol Phenylbutyrate Oral Liquid, 1.1 grams per mL. |
| 2026-03-04 | Immedica Pharma AB | Regulatory Ravicti® (glycerol phenylbutyrate) approved in United Arab Emirates and Sultanate of Oman immedica.com ↗
Immedica today announced that Ravicti® (glycerol phenylbutyrate) has received regulatory approval in the United Arab Emirates and the Sultanate of Oman, further expanding access to treatment for patients living with rare diseases in the region. |
| 2025-12-22 | Immedica Pharma AB | Regulatory Immedica and OrphanPacific announce approval of Ravicti® (glycerol phenylbutyrate) in Japan for the treatment of UCDs immedica.com ↗
Immedica and its partner OrphanPacific, Inc. announce that the Japanese Ministry of Health, Labour and Welfare (MHLW) has granted marketing authorization for Ravicti® (glycerol phenylbutyrate) for use as adjunctive therapy for the chronic management of patients with urea cycle disorders (UCDs). |
| 2025-08-18 | Immedica Pharma AB | Regulatory Ravicti® (glycerol phenylbutyrate) approved in Saudi Arabia, Qatar and Kuwait immedica.com ↗
Immedica is pleased to announce that Ravicti® (glycerol phenylbutyrate) has received regulatory approval in Saudi Arabia, Qatar, and Kuwait. |
| 2024-12-27 | Immedica Pharma AB | Regulatory Application to the Ministry of Health in Japan for Ravicti® (Glycerol Phenylbutyrate) submitted immedica.com ↗
Immedica is pleased to announce that its partner OrphanPacific, Inc has submitted an application to the Ministry of Health, Labour and Welfare in Japan for the approval of Ravicti® (glycerol phenylbutyrate) for the treatment of Urea Cycle Disorders (UCD). |
| 2024-03-21 | Immedica Pharma AB | Regulatory Ravicti® (glycerol phenylbutyrate) approved by the Taiwan Food and Drug Administration (TFDA) for the treatment of Urea Cycle Disorders immedica.com ↗
We are pleased to announce the approval of Ravicti in Taiwan, marking a significant milestone in our commitment to providing effective treatments for urea cycle disorders. |
| 2023-09-29 | Immedica Pharma AB | Regulatory Ravicti® (glycerol phenylbutyrate) approved by the National Administration of Drugs, Food and Medical Devices in Argentina for treatment of urea cycle disorders immedica.com ↗ |
| 2022-12-21 | Immedica Pharma AB | Regulatory Ravicti® (glycerol phenylbutyrate) approved by the Department of Health in Hongkong for the treatment of Urea Cycle Disorders immedica.com ↗
Immedica Pharma, a pharmaceutical company focused on commercialisation of rare and specialty care products, and Winhealth Pharma today announce that Ravicti® (Glycerol phenylbutyrate) has been approved by the Department of Health in Hongkong for the treatment of urea cycle disorders. |
| 2022-09-28 | Immedica Pharma AB | Regulatory NMPA Grants Priority Review to NDA for Glycerol Phenylbutyrate immedica.com ↗
Immedica Pharma AB is pleased to announce that RAVICTI® (glycerol phenylbutyrate), has been accepted and granted Priority Review for the New Drug Application (NDA) in China. |
| 2022-08-24 | Immedica Pharma AB | Regulatory Immedica Pharma and Innovative Medicines announces ANMAT and INVIMA filing acceptance of RAVICTI® (glycerol phenylbutyrate) for chronic management of patients with UCDs immedica.com ↗
Immedica Pharma AB, and Innovative Medicines SA, announces two submissions of marketing authorization applications to: The National Agency of Medicines, Food and Medical Technology (ANMAT) in Argentina. |
| 2019-01-30 | Sobi, Inc. | Regulatory Ravicti® now available for all ages in Europe following approval of the expanded paediatric indication sobi.com ↗ |
All press releases naming this drug 20 releases
Evidence & citations 14 cited values
Every value below carries the sentence it was read from. 27 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 201010“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 | 4-PBA | “Other Name(s): Ravicti; glycerol phenylbutyrate oral liquid; GPB; 4-phenylbutyrate; 4-PBA” NCT07847918 ↗ |
| Known as | 4-phenylbutyrate | “Other Name(s): Ravicti; glycerol phenylbutyrate oral liquid; GPB; 4-phenylbutyrate; 4-PBA” NCT07847918 ↗ |
| 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 ↗4“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 “Other Name(s): Ravicti; glycerol phenylbutyrate oral liquid; GPB; 4-phenylbutyrate; 4-PBA” NCT07847918 ↗ “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 NCT00999167 ↗ |
| 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 ↗8“Other Name(s): Ravicti; glycerol phenylbutyrate oral liquid; GPB; 4-phenylbutyrate; 4-PBA” NCT07847918 ↗ “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 ↗ |