Bile Acid Synthesis Defect
2
Pipeline Programs
1
Companies
1
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
0
0
1
1
Early DiscoveryClinical DevelopmentMarket
On Market (1)
Approved therapies currently available
Competitive Landscape
1 companies ranked by most advanced pipeline stage
Trial Timeline
Clinical trial activity over time
2021
2022
2023
2024
2025
2026
Mirum PharmaceuticalsCholic Acid
Clinical Trials (1)
Total enrollment: 53 patients across 1 trials
Open Label, Continuation Study of Cholic Acid in Subjects With Inborn Errors of Bile Acid Synthesis
Start: Jan 2010Est. completion: Jul 201653 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