RAVICTI (glycerol phenylbutyrate)
RAVICTI is a nitrogen-binding agent indicated for the chronic management of patients with urea cycle disorders (UCDs) who cannot be managed by dietary protein restriction or amino acid supplementation alone. It must be used in combination with dietary protein restriction and, in some cases, additional dietary supplements such as essential amino acids or arginine. The drug is not intended for the treatment of acute hyperammonemia because more rapidly acting interventions are required to reduce plasma ammonia levels. Furthermore, the safety and efficacy of RAVICTI have not been established for the treatment of N-acetylglutamate synthase (NAGS) deficiency.
How RAVICTI Works
RAVICTI is a triglyceride containing phenylbutyrate (PBA), which is metabolized into the active moiety phenylacetic acid (PAA). PAA conjugates with glutamine in the liver and kidneys to form phenylacetylglutamine (PAGN), which is then excreted by the kidneys. Because PAGN contains two molecules of nitrogen, this process provides an alternative vehicle for the excretion of waste nitrogen. This pathway helps prevent the accumulation of toxic levels of ammonia that occur when the enzymes or transporters necessary for the urea cycle are deficient.
Development Insights
Details
- Status
- Prescription
- First Approved
- 2013-02-01
- Patent Cliff
- 2030
- Routes
- ORAL
- Dosage Forms
- LIQUID
RAVICTI Approval History
What RAVICTI Treats
3 indicationsRAVICTI is approved for 3 conditions since its original approval in 2013. These indications span multiple therapeutic areas including oncology, immunology, and more.
- Urea Cycle Disorder
- Hyperammonemia
- N-Acetylglutamate Synthase Deficiency
RAVICTI 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 RAVICTI
3 of 6FDA-approved drugs for similar conditions. Compare mechanisms and indications to understand treatment alternatives.
Clinical Trial Registry
4 trials| Trial | Sponsor ID | Phase | Status | Title |
|---|---|---|---|---|
| NCT03335488 results posted | HPN-100-021 2015-000075-27 | Ph 4 | completed | Study of Glycerol Phenylbutyrate & Sodium Phenylbutyrate in Phenylbutyrate Naïve Patients With Urea Cycle Disorders (UCDs) |
| NCT02246218 results posted | HPN-100-009 2016-003460-38 | Ph 4 | completed | A Study of the Safety, Efficacy and Pharmacokinetics of Glycerol Phenylbutyrate in Pediatric Subjects Under 2 Years of Age With Urea Cycle Disorders |
| NCT02046434 | 13-2808 | Ph 1 | completed | Phenylbutyrate Response as a Biomarker for Alpha-synuclein Clearance From the Brain |
| NCT01881984 results posted | PRO13050530 | Ph 1 | completed | Use of Ravicti™ in Patients With MCAD Deficiency With the 985A>G (K304E) Mutation |
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.
RAVICTI FDA Label Details
Indications & Usage
FDA Label (PDF)RAVICTI is indicated for the treatment of Urea Cycle Disorder; Hyperammonemia; N-Acetylglutamate Synthase Deficiency.
RAVICTI Patents & Exclusivity
Patents (14 active)
Pro Intelligence Preview
Deep insights for RAVICTI
Revenue Insights
- • Quarterly revenue tracking
- • Historical trend analysis
Patent Timeline
- • Cliff: 2030
- • 14 active patents
Trial Analysis
- • 4 total trials
- • Stage: Mature
Competitive Landscape
- • 6 similar drugs
- • Same target/indication analysis
Full approval history • All patents • Revenue trends • Competitor analysis
Data Sources
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