Long-chain Fatty Acid Oxidation Disorders (LC-FAOD)
0
Pipeline Programs
1
Companies
2
Clinical Trials
0
Approved Products
Pipeline by Development Stage
Preclinical
Phase 1
Phase 1/2
Phase 2
Phase 2/3
Phase 3
On Market
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0
0
0
0
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Early DiscoveryClinical DevelopmentMarket
Competitive Landscape
1 companies ranked by most advanced pipeline stage
Ultragenyx PharmaceuticalNOVATO, CA
2 programsUX007PHASE_21 trial
TriheptanoinPHASE_31 trial
Active Trials
Trial Timeline
Clinical trial activity over time
2021
2022
2023
2024
2025
2026
2027
Ultragenyx PharmaceuticalTriheptanoin
Ultragenyx PharmaceuticalUX007
Clinical Trials (2)
Total enrollment: 98 patients across 2 trials
A Study to Determine the Effect of Triheptanoin Compared With Even-Chain MCT on MCEs in Pediatric Patients With LC-FAOD
Start: Feb 2023Est. completion: Aug 202769 patients
Phase 3Active Not Recruiting
A Study of UX007 (Triheptanoin) in Participants With Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD)
Start: Feb 2014Est. completion: Aug 201629 patients
Phase 2Completed
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
1 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.