Glycerol phenylbutyrate
Approved · FDA / EMAAlso known as GPB, GT4P, Ravicti, Glyceryl Tri-4-Phenylbutyrate.
Recent clinical, regulatory, research and industry developments relating to this drug.
Approval: GLYCEROL PHENYLBUTYRATE (ANDA220579)
Phenylbutyrate Response As a Biomarker for Alpha-Synuclein Clearance From Brain
Profile
Canonical identifiers, marketed brand names and mechanism, resolved across RxNorm, ChEMBL and ATC.
Regulatory timeline
The complete regulatory record, grouped by authority — approvals, safety advisories and label changes. Each authority shows its most recent events; expand one to read its full history.
Indication: Ravicti is indicated for use as adjunctive therapy for chronic management of patients with urea cycle disorders (UCDs) including deficiencies of carbamoyl… Show full indicationShow less
Ravicti is indicated for use as adjunctive therapy for chronic management of patients with urea cycle disorders (UCDs) including deficiencies of carbamoyl phosphate-synthase-I (CPS), ornithine carbamoyltransferase (OTC), argininosuccinate synthetase (ASS), argininosuccinate lyase (ASL), arginase I (ARG) and ornithine translocase deficiency hyperornithinaemia-hyperammonaemia homocitrullinuria syndrome (HHH) who cannot be managed by dietary protein restriction and/or amino acid supplementation alone. Ravicti must be used with dietary protein restriction and, in some cases, dietary supplements (e.g., essential amino acids, arginine, citrulline, protein-free calorie supplements).
Data from the U.S. Food and Drug Administration (U.S. Food and Drug Administration), public domain (CC0).
Contains information from the European Medicines Agency (European Medicines Agency), © EMA, reused under CC BY 4.0.
Clinical trials
The current development programme across all trial phases — status mix, phase distribution and the late-stage studies shaping the evidence base.
Late-stage studies
Phase III+ trials still open or recently active — where late-stage evidence is being generated.
Recent completions
Trials that read out recently, adding to the completed evidence base.
Research activity
Key research shaping understanding of this drug, combining the latest publications with the most influential evidence.
Journals, researchers & institutions
- Molecular genetics and metabolism2
- Berry SA2
- Diaz GA2
- Ficicioglu C2
- Harding CO2
- Lichter-Konecki U2
- Longo N2
- McCandless SE2
- Robinson B2
- Children's Hospital of Philadelphia2
- Children's Hospital of Pittsburgh2
- Icahn School of Medicine at Mount Sinai2
- Maine Medical Center2
- Oregon Health & Science University2
- University of Florida2
- RxNorm (U.S. National Library of Medicine) — drug identity
- ChEMBL (EMBL-EBI) & UniProt — pharmacology and targets
- Europe PMC — research literature
- ClinicalTrials.gov — clinical trials
- Regulatory event sources are credited in the Regulatory Timeline above.