Elamipretide Hydrochloride
Elamipretide Hydrochloride is a mitochondria-targeting peptide under investigation for the treatment of rare mitochondrial disorders and age-related conditions. It works by protecting mitochondrial function and reducing cellular stress. Clinical trials have explored its use in conditions affecting tissues with high energy demands, such as the heart, eyes, and skeletal muscles.
Elamipretide Hydrochloride is an investigational mitochondrial-protective peptide being studied for rare genetic disorders and conditions affecting high-energy tissues.
Key facts at a glance
- RxNorm
- 2725599
- Generic
- Elamipretide Hydrochloride
- Class
- pin-variant
- Brands
- —
- Availability
- rx
- Lactation
- unknown
- Routes
- —
- Forms
- —
- Regions
- US, EU, CN, JP
- Monitoring
- —
This page is for informational purposes only.
Do not use this information to diagnose yourself or to replace a physician's advice. Always consult a qualified healthcare provider before starting, stopping, or changing any medication. If you believe you are experiencing a medical emergency, call your local emergency number immediately.
Under investigation primarily for primary mitochondrial myopathy (a rare genetic disorder affecting muscle function), geographic atrophy (an advanced form of dry age-related macular degeneration), and heart failure with reduced ejection fraction. Not yet FDA-approved for any indication as of the available data.
Elamipretide is a cell-permeable peptide that selectively targets and accumulates within the inner mitochondrial membrane. It is designed to protect mitochondrial structure, reduce oxidative stress, and improve mitochondrial function in tissues with high energy demands.
This medication is currently investigational and not FDA-approved. Reported side effects in clinical trials have included mild to moderate reactions at injection sites. Patients should only use this under supervision in clinical trial settings or as prescribed off-label by a qualified healthcare provider. Efficacy and safety profiles are still being established through ongoing research.
Official drug label
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This information is for educational purposes only and is not a substitute for professional medical advice, diagnosis, or treatment.
This content is for general informational and educational purposes only and is not a substitute for professional medical advice, diagnosis, or treatment. Always seek the guidance of a qualified healthcare provider with any questions you have about a medical condition, and never disregard professional medical advice or delay seeking it because of something you read here.
If you think you may have a medical emergency, call your local emergency number or go to the nearest emergency department immediately.
Medically reviewed by WellAlly Editorial Team Last reviewed August 28, 2026. · Read our editorial policy