Elamipretide: First Approval

<p dir="ltr"><b>Funding</b> The preparation of this review was not supported by any external funding.</p><p dir="ltr"><b>Declarations</b></p><p dir="ltr"><b>Authorship and Conflict of interest</b> During the peer review process the manufacturer of the agent under review was offered an opportunity to comment on the article. Changes resulting from any comments received were made by the author on the basis of scientific completeness and accuracy. Matt Shirley is a salaried employee of Adis International Ltd/Springer Nature, and declares no relevant conflicts of interest. All authors contributed to this article and are responsible for its content.</p><p dir="ltr"><b>Ethics approval, Consent to participate, Consent to publish, Availability of data and material, Code availability </b>Not applicable.</p><p dir="ltr">Additional information about this Adis Drug Review can be found <a href="http://www.springer.com/gp/adis/products-services/adis-journals-newsletters/adis-drug-reviews" rel="noreferrer noopener" target="_blank">here</a>.</p><p dir="ltr"><b>Abstract</b></p><p dir="ltr">Elamipretide (Forzinity™) is a mitochondrial cardiolipin binder being developed by Stealth BioTherapeutics for the treatment of a range of disorders featuring mitochondrial dysfunction. In September 2025, elamipretide was granted accelerated approval in the USA for use to improve muscle strength in adult and pediatric patients with Barth syndrome weighing ≥ 30 kg. With this accelerated approval, elamipretide became the first disease-specific treatment approved for Barth syndrome, an ultra-rare X-linked recessive genetic disorder. Elamipretide is also under phase III clinical development for use in the treatment of dry age-related macular degeneration and mitochondrial myopathies. This article summarizes the milestones in the development of elamipretide leading to this first approval for Barth syndrome.</p><p dir="ltr">© The Author(s), under exclusive licence to Springer Nature Switzerland AG 2025</p>