Mitochondrial Myopathies
2
Pipeline Programs
5
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
1
0
1
0
Early DiscoveryClinical DevelopmentMarket
Drug Modality Breakdown
Peptide
2100%
+ 2 programs with unclassified modality
Competitive Landscape
4 companies ranked by most advanced pipeline stage
Trial Timeline
Clinical trial activity over time
2021
2022
2023
2024
2025
2026
BioTherapeutics IncElamipretide
BiogenOmaveloxolone capsules, 2.5 mg
Clinical Trials (2)
Total enrollment: 155 patients across 2 trials
Study to Evaluate Efficacy and Safety of Elamipretide in Subjects With Primary Mitochondrial Disease From Nuclear DNA Mutations (nPMD)
Start: Apr 2022Est. completion: Dec 2024102 patients
Phase 3Completed
RTA 408 Capsules in Patients With Mitochondrial Myopathy - MOTOR
Start: May 2015Est. completion: Nov 201753 patients
Phase 2Completed
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
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.