Elamipretide: Uses, Mechanism, and Clinical Evidence

For patients and families living with Barth syndrome, a rare genetic disorder that weakens skeletal and heart muscle, treatment options have historically been limited. Elamipretide, sold under the brand name FORZINITY, is now approved to address one part of that unmet need.

Key takeaways

• Elamipretide is a mitochondrial cardiolipin binder marketed as FORZINITY.
• It is approved to improve muscle strength in adult and pediatric Barth syndrome patients weighing at least 30 kg.
• The approval is an accelerated approval based on knee extensor muscle strength, an intermediate endpoint, with continued approval contingent on a confirmatory trial.
• At least 21 clinical trials have studied elamipretide across different conditions, including a completed Phase 2 heart failure study and an active Phase 3 dry AMD trial.
• No boxed warning is present on the label.

What Elamipretide Is

Elamipretide is classified as a mitochondrial cardiolipin binder. It works by localizing to the inner mitochondrial membrane, where it helps improve mitochondrial morphology and function. Because mitochondria supply energy to muscle cells, supporting their structure is the basis for the drug's effect on muscle strength.

What Elamipretide Is Approved For

FORZINITY is indicated to improve muscle strength in adult and pediatric patients with Barth syndrome who weigh at least 30 kg. This indication was granted under accelerated approval, based on improvement in knee extensor muscle strength rather than a confirmed long-term clinical benefit. Continued approval for this use may depend on verifying clinical benefit in a follow-up confirmatory trial. The label does not carry a boxed warning.

What the Evidence Shows

Elamipretide has been studied in at least 21 registered clinical trials spanning multiple conditions beyond its approved use. A completed Phase 2 trial evaluated subcutaneous elamipretide's effect on left ventricular function in people with stable heart failure with preserved ejection fraction. A separate completed Phase 2 study examined its impact on skeletal muscle function in elderly participants. A Phase 3 trial, still active and not recruiting, is assessing the efficacy, safety, and pharmacokinetics of subcutaneous elamipretide in people with dry age-related macular degeneration.