Is Elamipretide safe?
Few hundred people; injection-site reactions common
Injection-site reactions are the main side effect. They include pain, itching, redness and hardening. On the label, all 12 TAZPOWER patients had a local reaction while on elamipretide. These were also the commonest adverse events in the open-label extension. The label also notes a temporary rise in eosinophils, a type of white blood cell. In MMPOWER-3 the drug was described as well tolerated. Most adverse events were mild to moderate. Only a few hundred people in total have used it. So rare effects cannot be ruled out. 124
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Citations
- Thompson 2024, Genetics in Medicine: 168-week open-label extension of TAZPOWER, elamipretide in Barth syndrome doi.org
- Forzinity (elamipretide) prescribing information drugs.com
- 2025, Orphanet J Rare Dis: psychometric evaluation of the Barth Syndrome Symptom Assessment in the TAZPOWER trial pubmed.ncbi.nlm.nih.gov
- Karaa 2023, Neurology: efficacy and safety of elamipretide in primary mitochondrial myopathy (MMPOWER-3) pubmed.ncbi.nlm.nih.gov
Reference information only, not medical advice. Drawn from the Elamipretide article, fact-checked against the cited sources. No dosing anywhere on this site.