Long-chain Fatty Acid Oxidation Disorders (LC-FAOD)
3
Pipeline Programs
1
Companies
2
Clinical Trials
1
Approved Products
Pipeline by Development Stage
Preclinical
Phase 1
Phase 1/2
Phase 2
Phase 2/3
Phase 3
On Market
0
0
0
1
0
1
1
Early DiscoveryClinical DevelopmentMarket
On Market (1)
Approved therapies currently available
Competitive Landscape
1 companies ranked by most advanced pipeline stage
Ultragenyx PharmaceuticalNOVATO, CA
2 programs1
1
1
TriheptanoinPhase 31 trial
UX007Phase 21 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 late-stage (Phase 3) programs — potential near-term approvals
1 companies competing in this space