Delivery 7–10 daysForzinity (elamipretide) 280 mg vials for injection
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Description
💊 Prescription cardiolipin-binding peptide indicated to improve muscle strength in adult and paediatric patients with Barth syndrome weighing at least 30 kg.
▪︎ Elamipretide 280 mg/3.5 mL (80 mg/mL), carton of 4 single-patient-use vials; 40 mg subcutaneously once daily at the same time each day
▪︎ Manufacturer: Stealth BioTherapeutics
▪︎ FDA approval: September 2025
🔬 How it works: the inner mitochondrial membrane is held in shape by cardiolipin, a wedge-shaped lipid that keeps the respiratory chain complexes in working position. In Barth syndrome a fault in the TAZ gene disables tafazzin, the enzyme that matures cardiolipin, so an intermediate form accumulates and the membrane loses its geometry. Elamipretide is a four-amino-acid peptide that binds cardiolipin and stabilises that geometry — it does not repair the gene and does not create normal cardiolipin, it braces a structure whose internal fastening has worn out. 📊 Evidence: in the randomised crossover part of TAZPOWER (Genet Med 2021) in 12 patients both primary endpoints were missed. In the open-label extension walking distance improved by 95.9 m at week 36 and by 96.1 m at week 168 (Genet Med 2024). Against 19 untreated patients matched by propensity score (Orphanet J Rare Dis 2022) the difference was 79.7 m at week 64 and 91.0 m at week 76, with muscle strength 40.8 and 56.7 newtons greater; left ventricular stroke volume rose in the treated and fell in the untreated.
⚠️ Prescription-only, prescribed by a specialist centre; genetic confirmation of the diagnosis is required. Approval is accelerated on an intermediate endpoint (knee extensor strength) and the indication is narrow — to improve muscle strength, not to treat the syndrome; a confirmatory trial is pending. The product contains benzyl alcohol and must never be used in neonates, and intravenous administration is not approved. Not established below 30 kg; the dose is reduced in severe renal impairment. In a large randomised trial in primary mitochondrial myopathy the same drug showed no effect.
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