TheraRadar
Data updated: May 26, 2026

OLPRUVA (sodium phenylbutyrate)

Rare Disease Approved 2022-12-22

OLPRUVA is indicated for the treatment of Urea Cycle Disorder; Hyperammonemic Encephalopathy.

2
Indications
--
Phase 3 Trials
3
Years on Market

Details

Status
Prescription
First Approved
2022-12-22
Patent Cliff
2036

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Routes
ORAL
Dosage Forms
FOR SUSPENSION

Companies

Active Ingredient: SODIUM PHENYLBUTYRATE

OLPRUVA Approval History

2023
2024
2025
2026
Original
New Indication
New Form
Label Update
16 FDA actions from 2022 to 2025 · 1 indication expansions
Oct 2025 SUPPL
Efficacy
Dec 2022 ORIGINAL
Update · Type 5 - New Formulation or New Manufacturer

What OLPRUVA Treats

2 indications

OLPRUVA is approved for 2 conditions since its original approval in 2022. These indications span multiple therapeutic areas including oncology, immunology, and more.

  • Urea Cycle Disorder
  • Hyperammonemic Encephalopathy
Source: FDA Label

OLPRUVA Competitive Set

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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 OLPRUVA

3 of 4

FDA-approved drugs for similar conditions. Compare mechanisms and indications to understand treatment alternatives.

SODIUM PHENYLBUTYRATE
SODIUM PHENYLBUTYRATE
2 shared
NAVINTA LLC
Shared indications:
Urea Cycle DisorderHyperammonemic Encephalopathy
PHEBURANE
SODIUM PHENYLBUTYRATE
1 shared
MEDUNIK
Shared indications:
Urea Cycle Disorder
RAVICTI
GLYCEROL PHENYLBUTYRATE
1 shared
HORIZON THERAP US
Shared indications:
Urea Cycle Disorder
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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
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Active Pipeline

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Key Completed Trials

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Completed studies with published results, ranked by significance

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Trial Timeline

Full development history with FDA approval milestones

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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.

OLPRUVA FDA Label Details

Indications & Usage

FDA Label (PDF)

OLPRUVA is indicated for the treatment of Urea Cycle Disorder; Hyperammonemic Encephalopathy.

View full patent landscape →
3 OB patents · 1 families · 53 international docs across 23 countries

OLPRUVA Patents & Exclusivity

Latest Patent: Oct 2036

Patents (3 active)

US11202767 Expires Oct 17, 2036
US11433041 Expires Oct 17, 2036
US11154521 Expires Oct 17, 2036
Source: FDA Orange Book

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Revenue Insights

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Patent Timeline

  • Cliff: 2036
  • 192 active patents

Trial Analysis

  • Clinical trial tracking
  • Development stage analysis

Competitive Landscape

  • 4 similar drugs
  • Same target/indication analysis
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Data Sources

Data sourced from official FDA and NIH databases. Click links to verify on original sources.