Study summary · research use only
Elamipretide: First Approval
Plain-language summary
Paraphrased from the published abstract below — not a verdict on whether anything works.
This article (species not specified; human regulatory context) summarizes the development milestones leading to the first approval of elamipretide (Forzinity), a mitochondrial cardiolipin binder developed by Stealth BioTherapeutics for disorders involving mitochondrial dysfunction. In September 2025, elamipretide received accelerated approval in the USA to improve muscle strength in adult and pediatric patients with Barth syndrome weighing ≥ 30 kg, becoming the first disease-specific treatment approved for this ultra-rare X-linked recessive genetic disorder. The article notes elamipretide is also in phase III clinical development for dry age-related macular degeneration and mitochondrial myopathies.
Abstract
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.
pepmg summarizes the peer-reviewed literature and links to every source — it sells nothing, ships nothing, and gives no medical, dosing, or human-use guidance. Don't just trust this summary: follow the citation to its source and read it yourself. Research use only.