RAVICTI
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.
Details
- Status
- Prescription
- First Approved
- 2013-02-01
- 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
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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
ProIndications & Usage
FDA Label (PDF)RAVICTI is indicated for use as a nitrogen-binding agent for chronic management of patients with urea cycle disorders (UCDs) who cannot be managed by dietary protein restriction and/or amino acid supplementation alone. RAVICTI must be used with dietary protein restriction and, in some cases, dietary supplements (e.g., essential amino acids, arginine, citrulline, protein-free calorie supplements). Limitations of Use: RAVICTI is not indicated for the treatment of acute hyperammonemia in patients with UCDs because more rapidly acting interventions are essential to reduce plasma ammonia levels. Th...
RAVICTI Patents & Exclusivity
Patents (14 active)
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Data Sources
Data sourced from official FDA and NIH databases. Click links to verify on original sources.