PHEBURANE (sodium phenylbutyrate)
PHEBURANE is indicated for the treatment of Urea Cycle Disorder.
How PHEBURANE Works
Sodium phenylbutyrate is a prodrug that is metabolized to phenylacetate. Phenylacetate conjugates with glutamine via acetylation to form phenylacetylglutamine, which is then excreted by the kidneys. This process provides an alternative pathway for waste nitrogen excretion, bypassing the deficient enzymes of the urea cycle.
Details
- Status
- Prescription
- First Approved
- 2022-06-17
- Routes
- ORAL
- Dosage Forms
- PELLETS
PHEBURANE Approval History
What PHEBURANE Treats
1 indicationsPHEBURANE is approved for 1 conditions since its original approval in 2022. These indications span multiple therapeutic areas including oncology, immunology, and more.
- Urea Cycle Disorder
PHEBURANE Competitive Set
ProThree rings of competition based on shared molecular targets and treated indications.
Indication competitors
Same indication, different mechanism — what else might this patient receive?
Filters applied: drops same-active-ingredient (505(b)(2) reformulations), route-mismatch (topical vs systemic), and cross-therapeutic-area matches in same-indication rings.
Drugs Similar to PHEBURANE
3 of 4FDA-approved drugs for similar conditions. Compare mechanisms and indications to understand treatment alternatives.
Clinical Trial Registry
9 trials| Trial | Sponsor ID | Phase | Status | Title |
|---|---|---|---|---|
| NCT06069375 | STUDY23060034 | Ph 2 | suspended | Study of Sodium Phenylbutyrate (ACER-001) for the Treatment of Patients With Medium Chain Acyl-CoA Dehydrogenase Deficiency (MCADD) |
| NCT07125066 | STUDY25060137 | Ph 1 | enrolling by invitation | An Individual Patient, Open Label Study to Use ACER-001 to Treat Combined D,L-2 Hydroxyglutaric Aciduria (C-2HGA) |
| NCT06773026 | STUDY24100064 | Ph 2 | recruiting | Study of Sodium Phenylbutyrate (ACER-001) for the Treatment of Pediatric and Adults Patients With Medium Chain Acyl-CoA Dehydrogenase Deficiency (MCADD) |
| NCT05349435 | PBI-4050-CT-9-21 | Ph 1 | terminated | A Healthy Volunteer Study to Compare Fezagepras (PBI-4050) With Sodium Phenylbutyrate |
| NCT03734263 TIGEM2-PDH | TIGEM2-PDH | Ph 2 | completed | Use of Phenylbutyrate Therapy for Patients With Pyruvate Dehydrogenase Complex Deficiency. |
| NCT00345605 results posted | RDCRN 5102 U54HD061221 | Ph 2 | completed | Arginine and Buphenyl in Patients With Argininosuccinic Aciduria (ASA), a Urea Cycle Disorder |
| NCT01096095 | CONEP CAAE 0527.0.001.000-07 | Ph 2 | withdrawn | Pilot Study of Safety and Efficacy of Sodium Phenylbutyrate in Spinocerebellar Ataxia Type 3 |
| NCT00439218 NPTUNE 02 results posted | N01NS42361_NPTUNE02 HHSN265200423611C, N01NS42361 | Ph 1, Ph 2 | terminated | Clinical Trial of Sodium Phenylbutyrate in Children With Spinal Muscular Atrophy Type I |
| NCT00439569 NPTUNE01 results posted | N01NS42361_NPTUNE01 HHSN265200423611C, N01NS42361 | Ph 1, Ph 2 | terminated | Clinical Trial of Sodium Phenylbutyrate in Children With Spinal Muscular Atrophy Types II or III |
Active Pipeline
Ongoing clinical trials by development phase
Key Completed Trials
Completed studies with published results, ranked by significance
Trial Timeline
Full development history with FDA approval milestones
Understanding FDA Approval Types
| Count | Type | What it means |
|---|---|---|
| - | ORIG | Original approval - drug first enters market |
| - | SUPPL - Efficacy | New indication (new disease/condition approved) |
| - | SUPPL - Labeling | Label text changes (warnings, dosing updates) |
| - | SUPPL - Manufacturing | Production changes (new facility) |
| - | SUPPL - Chemistry | Formulation changes (new dosage strength) |
Green lines in the timeline show ORIG and Efficacy approvals - the clinically meaningful milestones.
PHEBURANE FDA Label Details
Indications & Usage
FDA Label (PDF)PHEBURANE is indicated for the treatment of Urea Cycle Disorder.
Track PHEBURANE with TheraRadar Pro
Watchlist alerts, full database access, CSV exports across 14,000+ drugs.
Data Sources
Data sourced from official FDA and NIH databases. Click links to verify on original sources.