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Urea Cycle Disorder

2
Pipeline Programs
4
Companies
3
Clinical Trials
0
Approved Products

Pipeline by Development Stage

Preclinical
Phase 1
Phase 1/2
Phase 2
Phase 2/3
Phase 3
On Market
0
0
0
0
0
0
2
Early DiscoveryClinical DevelopmentMarket

Competitive Landscape

2 companies ranked by most advanced pipeline stage

Amgen
AmgenTHOUSAND OAKS, CA
2 programs
2
RAVICTIPhase 41 trial
RAVICTIPhase 41 trial
Active Trials
NCT03335488Completed16Est. Dec 2022
NCT02246218Completed27Est. Jul 2017
Colorado Therapeutics
Colorado TherapeuticsCO - Louisville
1 program
Liver Disease in Urea Cycle DisordersN/A1 trial
Active Trials
NCT04612764Active Not Recruiting62Est. Dec 2026

Trial Timeline

Clinical trial activity over time

2021
2022
2023
2024
2025
2026
AmgenRAVICTI
AmgenRAVICTI
Colorado TherapeuticsLiver Disease in Urea Cycle Disorders

Clinical Trials (3)

Total enrollment: 105 patients across 3 trials

NCT03335488AmgenRAVICTI

Study of Glycerol Phenylbutyrate & Sodium Phenylbutyrate in Phenylbutyrate Naïve Patients With Urea Cycle Disorders (UCDs)

Start: Feb 2018Est. completion: Dec 202216 patients
Phase 4Completed
NCT02246218AmgenRAVICTI

A Study of the Safety, Efficacy and Pharmacokinetics of Glycerol Phenylbutyrate in Pediatric Subjects Under 2 Years of Age With Urea Cycle Disorders

Start: Dec 2014Est. completion: Jul 201727 patients
Phase 4Completed
NCT04612764Colorado TherapeuticsLiver Disease in Urea Cycle Disorders

Liver Disease in Urea Cycle Disorders

Start: Nov 2021Est. completion: Dec 202662 patients
N/AActive Not Recruiting

Phase Legend

Preclinical— Lab & animal studies
Phase 1— Safety & dosing
Phase 2— Efficacy testing
Phase 3— Large-scale trials
On Market— Approved & available

Key Insights

4 companies competing in this space

The information on this page is for informational purposes only and should not be used as a substitute for professional medical advice. Drug information is sourced from FDA, DailyMed, and other government databases. Adverse event data from FAERS does not establish causation. Always consult a healthcare professional for medical decisions.