Elamipretide
Also known as Forzinity · SS-31 · MTP-131 · Bendavia
The approved indication is improvement of muscle strength in people with Barth syndrome who meet label criteria. A separate phase 3 trial in primary mitochondrial myopathy did not improve its walking or fatigue primary endpoints.
- Evidence maturity
- Developing record
- Regulatory status
- FDA approved
- Recorded outcomes
- 3
- Indexed sources
- 3
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Summary
A mitochondria-targeting tetrapeptide with accelerated FDA approval for a narrow Barth syndrome indication.
What is Elamipretide being studied for?
Barth syndrome and mitochondrial research
How is it thought to work?
Binds cardiolipin and localizes to the inner mitochondrial membrane, where it is intended to support mitochondrial structure and function.
Recorded outcomes
3 graded outcomes and 3 sources for Elamipretide.
The graded outcome table, how each grade was decided, the regulatory detail, and every source with its link and identifier open with a free account. No payment details required.
Update history
- Editorial review of the profile and its sources.
- Record last updated in the Senzu database.
- Profile first published.