TheraRadar
Data updated: May 26, 2026

RELYVRIO (sodium phenylbutyrate)

Trial Activity: Stable 2 active trials
Orphan Drug Priority Review
Rare Disease Approved 2022-09-29
Jump to: Indications Approvals Trials Competitors Safety Patents

Development Insights

Jerry Vockley, MD, PhD conducting 3 trials (33%)
11 indications explored (Broad Platform)
→ medium-chain acyl-coa dehydrogenase deficiency (2 trials)
→ combined d,l-2-hydroxyglutaric aciduria (1 trials)
→ hyperammonemia (1 trials)
1
Indication
--
Phase 3 Trials
1
Priority Reviews
3
Years on Market

Details

Status
Discontinued
First Approved
2022-09-29
Patent Cliff
2040

Pro Metrics

Patent cliff and revenue data

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Routes
ORAL
Dosage Forms
FOR SUSPENSION

Companies

AMYLYX
Active Ingredient: SODIUM PHENYLBUTYRATE , TAURURSODIOL

RELYVRIO Approval History

2023
2024
2025
2026
Original
New Indication
New Form
Label Update
1 FDA actions from 2022 to 2022
Sep 2022 ORIGINAL Priority
New Drug · Type 1 - New Molecular Entity
FDA Letter Label

What RELYVRIO Treats

1 FDA approvals

Originally approved for its first indication in 2022 .

  • Other (1)
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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NCT01096095 CONEP CAAE 0527.0.001.000-07 Ph 2 withdrawn Pilot Study of Safety and Efficacy of Sodium Phenylbutyrate in Spinocerebellar Ataxia Type 3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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Ongoing clinical trials by development phase

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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

ClinicalTrials.gov ↗ Pro

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.

RELYVRIO FDA Label Details

View full patent landscape →
5 OB patents · 2 families · 98 international docs across 25 countries

RELYVRIO Patents & Exclusivity

Latest Patent: Jul 2040
Exclusivity: Sep 2029

Patents (5 active)

US11583542 Expires Jul 27, 2040
US10857162 Expires Dec 24, 2033
US10251896 Expires Dec 24, 2033
US9872865 Expires Dec 24, 2033
US11071742 Expires Dec 24, 2033

Exclusivity

NCE Until Sep 2027
ODE-411 Until Sep 2029
Source: FDA Orange Book

Pro Intelligence Preview

Deep insights for RELYVRIO

Revenue Insights

  • • Quarterly revenue tracking
  • • Historical trend analysis

Patent Timeline

  • • Cliff: 2040
  • • 5 active patents

Trial Analysis

  • • 9 total trials
  • • Stage: Stable

Competitive Landscape

  • • Competitor tracking
  • • Same target/indication analysis
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Full approval history • All patents • Revenue trends • Competitor analysis

Data Sources

FDA Approval: NDA216660 → Patents: FDA Orange Book → Trials: ClinicalTrials.gov →

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

How We Calculate These Metrics

Trial Activity Stage

Measures the current development activity pattern based on trial phases, status, and trends. Important: This measures R&D activity, not commercial lifecycle.

Trial statuses: "Active" means recruiting or ongoing. "Completed" means reached planned endpoint. "Terminated" means stopped early—often due to safety, efficacy, or business reasons.

  • Growth: High proportion of early-phase trials (Phase 1/2), active development
  • Expansion: Significant Phase 3 activity, approaching or pursuing approvals
  • Mature: Substantial Phase 4 post-marketing studies
  • Stable: Mixed phase distribution, steady development
  • Declining: Low active trial ratio, reduced R&D investment