Monocarboxylate Transporter 8 Deficiency

1
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
0
0
0
0
0
1
0
Early DiscoveryClinical DevelopmentMarket

Competitive Landscape

1 companies ranked by most advanced pipeline stage

Egetis Therapeutics
Egetis TherapeuticsSweden - Stockholm
2 programs
1
TiratricolPhase 31 trial
TiratricolN/A1 trial
Active Trials
NCT05911399Available
NCT05579327CompletedEst. Sep 2025

Trial Timeline

Clinical trial activity over time

2023
2024
2025
2026
Egetis TherapeuticsTiratricol

Clinical Trials (2)

Withdrawal of Tiratricol Treatment in Males With Monocarboxylate Transporter 8 Deficiency (MCT8 Deficiency)

Start: Jul 2023Est. completion: Sep 2025
Phase 3Completed

Expanded Access Program for Tiratricol in Patients With Monocarboxylate Transporter 8 Deficiency

N/AAvailable

Phase Legend

PreclinicalLab & animal studies
Phase 1Safety & dosing
Phase 2Efficacy testing
Phase 3Large-scale trials
On MarketApproved & available

Key Insights

1 late-stage (Phase 3) programs — potential near-term approvals
1 companies competing in this space