SODIUM PHENYLBUTYRATE · NAVINTA LLC
Sodium phenylbutyrate tablets are indicated as adjunctive therapy in the chronic management of patients with urea cycle disorders involving deficiencies of carbamylphosphate synthetase (CPS), ornithine transcarbamylase (OTC), or argininosuccinic acid synthetase (AS). It is important that the diagnosis be made early and treatment initiated immediately to improve survival. Any episode of acute hyperammonemia should be treated as a life-threatening emergency.
Details
- Status
- Prescription
- First Approved
- 1996-04-30
- Routes
- ORAL
- Dosage Forms
- TABLET, POWDER
SODIUM PHENYLBUTYRATE Approval History
What SODIUM PHENYLBUTYRATE Treats
4 indicationsSODIUM PHENYLBUTYRATE is approved for 4 conditions since its original approval in 2011. These indications span multiple therapeutic areas including oncology, immunology, and more.
- Urea Cycle Disorders
- Carbamylphosphate Synthetase Deficiency
- Ornithine Transcarbamylase Deficiency
- Argininosuccinic Acid Synthetase Deficiency
SODIUM PHENYLBUTYRATE 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.
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.
SODIUM PHENYLBUTYRATE FDA Label Details
Indications & Usage
Sodium phenylbutyrate tablets are indicated as adjunctive therapy in the chronic management of patients with urea cycle disorders involving deficiencies of carbamylphosphate synthetase (CPS), ornithine transcarbamylase (OTC), or argininosuccinic acid synthetase (AS). It is indicated in all patients with neonatal-onset deficiency (complete enzymatic deficiency, presenting within the first 28 days of life). It is also indicated in patients with late-onset disease (partial enzymatic deficiency, presenting after the first month of life) who have a history of hyperammonemic encephalopathy. It is im...
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PHEBURANE
Full clinical data, patents, trials, and competitive landscape for sodium phenylbutyrate.
Data Sources
Data sourced from official FDA and NIH databases. Click links to verify on original sources.