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Mitochondrial Disease

2
Pipeline Programs
5
Companies
5
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
0
2
0
0
0
0
Early DiscoveryClinical DevelopmentMarket

Competitive Landscape

5 companies ranked by most advanced pipeline stage

Human BioSciences
Human BioSciencesWV - Martinsburg
2 programs
1
EPI-743Phase 1/2
Acute Infection in Mitochondrial Disease: Metabolism, Infection and ImmunityN/A
Genome & Company
Genome & CompanyKorea - Suwon
2 programs
1
EPI-743Phase 1/21 trial
Acute Infection in Mitochondrial Disease: Metabolism, Infection and ImmunityN/A1 trial
Active Trials
NCT04419870Recruiting400Est. May 2026
NCT01642056Completed20Est. Sep 2019
Rare Disease Therapeutics
1 program
Energy Requirements in Mitochondrial DiseaseN/A1 trial
Active Trials
NCT01776918Completed23Est. Sep 2014
Ribonova
RibonovaPA - Wynnewood
1 program
ProbucolN/A1 trial
Active Trials
NCT06931834Available
PTC Therapeutics
1 program
VatiquinoneN/A1 trial
Active Trials
NCT07159139No Longer Available

Trial Timeline

Clinical trial activity over time

2021
2022
2023
2024
2025
2026
Genome & CompanyEPI-743
Genome & CompanyAcute Infection in Mitochondrial Disease: Metabolism, Infection and Immunity
Rare Disease TherapeuticsEnergy Requirements in Mitochondrial Disease

Clinical Trials (5)

Total enrollment: 443 patients across 5 trials

NCT01642056Genome & CompanyEPI-743

EPI-743 for Metabolism or Mitochondrial Disorders

Start: Sep 2012Est. completion: Sep 201920 patients
Phase 1/2Completed
NCT06931834RibonovaProbucol

Expanded Access Program for R007 (Probucol) Tablets in Adults With Mitochondrial Disease and Chronic Kidney Disease

N/AAvailable
NCT07159139PTC TherapeuticsVatiquinone

Vatiquinone Expanded Access Protocol

N/ANo Longer Available
NCT04419870Genome & CompanyAcute Infection in Mitochondrial Disease: Metabolism, Infection and Immunity

Acute Infection in Mitochondrial Disease: Metabolism, Infection and Immunity

Start: Oct 2020Est. completion: May 2026400 patients
N/ARecruiting
NCT01776918Rare Disease TherapeuticsEnergy Requirements in Mitochondrial Disease

Energy Requirements in Mitochondrial Disease

Start: Feb 2012Est. completion: Sep 201423 patients
N/ACompleted

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 actively recruiting trials targeting 443 patients
5 companies competing in this space

The information on this page is for informational purposes only and should not be used as a substitute for professional medical advice. Drug information is sourced from FDA, DailyMed, and other government databases. Adverse event data from FAERS does not establish causation. Always consult a healthcare professional for medical decisions.