Elamipretide

What people use it for

• Barth syndrome (approved use)
• Mitochondrial disease and heart failure (research)
• Healthy ageing and energy (popular use)

What Elamipretide does in the body

A small peptide that travels to the inner membrane of mitochondria, the cell's power plants, and binds cardiolipin, a fat that holds the energy-producing machinery together. This is thought to stabilise that machinery, improve energy production and reduce oxidative stress.

Strength of evidence

Moderate for Barth syndrome (approved on surrogate endpoints); mixed results in other mitochondrial and heart conditions.

Look up the evidence

• Reviews on PubMed: Summaries of the published research
• Clinical trials on PubMed: Published studies in people
• ClinicalTrials.gov: Registered trials, past and ongoing
• Prescribing information (DailyMed, US): Official US label
• Patient leaflets (emc, UK): Official UK product information

Live searches of official databases. New to the terms? See the glossary.

Reported side effects & risks

• Injection-site reactions (very common)
• Headache
• Dizziness

Who should avoid it or take extra care

• Pregnant or breastfeeding (limited data)
• Anyone without the approved condition (Barth syndrome) outside a trial

Not a complete list. Check with a doctor or pharmacist about your own health and medicines.

Regulatory status

FDA-approved (accelerated approval) as Forzinity for Barth syndrome. Other uses are not approved.

How it's taken

Route: Injection under the skin once daily

How long it lasts (half-life): A few hours