Stages of Development for Elamipretide

The drug development process can be thought of as a series of stages, and successful drugs must pass through each stage to become available to patients.

Link to Stealth Biotherapeutics

In animal models, a single injection of the elamipretide was shown to reverse depressed mitochondrial energetics and to restore the cell redox environment in aged mouse skeletal muscle. Other studies demonstrated an effect in mouse models of neurodegeneration, cardiomyopathy, and age-related macular degeneration.

Preclinical safety and efficacy studies supported clinical development of elamipretide.

The Children’s Hospital of Philadelphia (CHOP) has a Phase 1/2 clinical trial studying the safety and efficacy of Elamipretide in the treatment of advanced stage FRDA symptoms, specifically vision loss and cardiac disease in both adults and older children. This study is completely enrolled and results are anticipated in the second half of 2024.

News and Press Releases

| Mar 28, 2022

Stealth BioTherapeutics Receives Orphan Drug Designation from FDA for Elamipretide for the Treatment of Friedreich's Ataxia

Industry News

Elamipretide Clinical Trials

Improve Mitochondrial Function & Reduce Oxidative Stress
Clinical Trial | Ages 16+

A Pilot Investigator Initiated Study to Evaluate the Safety, Tolerability and Efficacy of Elamipretide in the Treatment of Advanced Symptoms of Friedreich Ataxia (FRDA)

Children’s Hospital of Philadelphia
Phase I/II | In-person