Pyruvate Dehydrogenase Complex Deficiency
3
Pipeline Programs
3
Companies
3
Clinical Trials
1 recruiting
0
Approved Products
Pipeline by Development Stage
Preclinical
Phase 1
Phase 1/2
Phase 2
Phase 2/3
Phase 3
On Market
0
1
0
0
0
2
0
Early DiscoveryClinical DevelopmentMarket
Competitive Landscape
3 companies ranked by most advanced pipeline stage
Saol TherapeuticsGA - Roswell
2 programs1
DichloroacetatePhase 3
DichloroacetateN/A1 trial
Active Trials
NCT06931262Available
Trial Timeline
Clinical trial activity over time
2021
2022
2023
2024
2025
2026
2027
2028
2029
Medosome BiotecDichloroacetate
Ultragenyx PharmaceuticalTriheptanoin
Clinical Trials (3)
Total enrollment: 6 patients across 3 trials
Trial of Dichloroacetate in Pyruvate Dehydrogenase Complex Deficiency:
Start: Jul 2020Est. completion: Aug 2025
Phase 3Active Not Recruiting
Triheptanoin for Children With Primary-Specific Pyruvate Dehydrogenase Complex (PDC) Deficiency
Start: Jul 2024Est. completion: Jun 20296 patients
Phase 1Recruiting
Expanded Access Treatment Protocol With DCA for Patients With PDCD
N/AAvailable
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
2 late-stage (Phase 3) programs — potential near-term approvals
1 actively recruiting trials targeting 6 patients
3 companies competing in this space