CTX
1
Pipeline Programs
1
Companies
1
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
1
0
Early DiscoveryClinical DevelopmentMarket
Competitive Landscape
1 companies ranked by most advanced pipeline stage
Trial Timeline
Clinical trial activity over time
2021
2022
2023
2024
2025
2026
Mirum PharmaceuticalsBlinded CDCA 250 mg TID
Clinical Trials (1)
Total enrollment: 19 patients across 1 trials
Study to Evaluate Patients With Cerebrotendinous Xanthomatosis (RESTORE)
Start: Jan 2020Est. completion: Oct 202319 patients
Phase 3Completed
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