Forzinity®
Elamipretide is a mitochondrial cardiolipin binder.1
Elamipretide has the following uses:
Elamipretide is indicated to improve muscle strength in adult and pediatric patients with Barth syndrome weighing at least 30 kg.1
This indication is approved under accelerated approval based on an improvement in knee extensor muscle strength, an intermediate clinical endpoint.1 Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.1
Elamipretide hydrochloride is available in the following dosage form(s) and strength(s):
Injection: 280 mg of elamipretide per 3.5 mL (80 mg/mL) solution for injection in single-patient- use vials.1
Dosage in Adults and Pediatric Patients
Risk of Benzyl Alcohol Toxicity in Neonates
Elamipretide is not approved for use in neonates.1 Serious adverse reactions, including fatal reactions, of new onset or worsening metabolic acidosis that progressed to neurotoxicity, and in some cases gasping syndrome, have been reported in low-birth weight neonates (less than 2500 grams) and preterm neonates (gestational age less than 34 weeks) who received benzyl alcohol (BA)-containing drugs intravenously.1 Elamipretide is not approved for IV use.1 Gasping syndrome is a life-threatening condition in neonates caused by BA toxicity and is primarily characterized by multiorgan dysfunction secondary to metabolic acidosis, which leads to gasping respirations and death.1 The minimum amount of BA at which these serious adverse reactions, including fatal reactions, may occur is not known (elamipretide contains 20 mg of BA per mL).1
Hypersensitivity reactions, including serious allergic reactions requiring emergency medical intervention, have been reported in patients receiving elamipretide.1 These reactions have included skin manifestations such as rash, papular lesions, and eczematous dermatitis, as well as respiratory symptoms including cough.1 Reactions may occur within minutes to months after treatment initiation.1 Monitor patients for signs and symptoms of hypersensitivity reactions during treatment.1 If a serious hypersensitivity reaction occurs, do not administer further doses of elamipretide and institute appropriate emergency treatment, including epinephrine, antihistamines, and corticosteroids as clinically indicated.1 Patients who have experienced a serious hypersensitivity reaction should not be rechallenged with elamipretide.1 For mild to moderate skin reactions, consider treatment with topical corticosteroids and oral antihistamines.1 Consider discontinuation of elamipretide if persistent or severe skin reactions develop.1
Barth Syndrome is a rare, X-linked, recessive, genetic disorder and is not likely to affect females.1 Therefore, there are no data with elamipretide use in pregnant women to evaluate for a drug-related risk of major birth defects, miscarriage, or other adverse maternal or fetal outcomes.1 In animal reproduction studies, no adverse developmental outcomes occurred at any dose tested.1 Elamipretide contains benzyl alcohol as a preservative.1 Because benzyl alcohol is rapidly metabolized by a pregnant woman, benzyl alcohol exposure in the fetus is unlikely.1 The background risk of major birth defects and miscarriage for the indicated population is unknown.1 All pregnancies have a background risk of birth defect, loss, or other adverse outcomes.1 In the U.S.1 general population, the estimated background risk of major birth defects and miscarriage in clinically recognized pregnancies is 2 to 4% and 15 to 20%, respectively.1
Barth Syndrome is a rare, X-linked, recessive, genetic disorder and is not likely to affect females.1 Therefore, there is no data to evaluate the presence of elamipretide in human milk, the effect on the breastfed infant, or the effects on milk production.1 Elamipretide contains benzyl alcohol.1 Because benzyl alcohol is rapidly metabolized by a lactating female, benzyl alcohol exposure in the breastfed infant is unlikely.1 The developmental and health benefits of breastfeeding should be considered along with the mother's clinical need for elamipretide and any potential adverse effects on the breastfed infant from the drug or from the underlying maternal condition.1
The safety and effectiveness of elamipretide to improve muscle strength have been established in pediatric patients with Barth syndrome weighing at least 30 kg.1 Use of elamipretide for this indication is supported by improvement in knee extensor muscle strength, an intermediate clinical endpoint, observed in an open-label extension study of elamipretide that included 7 pediatric patients 12 years of age and older.1 The safety and effectiveness of elamipretide have not been established in pediatric patients weighing less than 30 kg.1 Elamipretide is not approved for use in neonates.1 Serious adverse reactions, including fatal reactions, of new onset or worsening metabolic acidosis that progressed to neurotoxicity, and in some cases gasping syndrome, have been reported in low-birth weight neonates and preterm neonates who received benzyl alcohol (BA)-containing drugs intravenously (elamipretide is not approved for IV use).1 Gasping syndrome is a life-threatening condition in neonates caused by BA toxicity that is characterized by new onset or worsening metabolic acidosis with gradual neurological deterioration, seizures, intracranial hemorrhage, hematologic abnormalities, skin breakdown, hepatic and renal failure, hypotension, bradycardia, and gasping respirations followed by death.1 In reported cases, BA in amounts of 99 to 234 mg/kg/day produced blood BA levels of 6.6 to 14.9 mg/dL, but the minimum amount of BA at which gasping syndrome may occur in neonates is not known (elamipretide contains 20 mg of BA per mL).1
Clinical studies of elamipretide did not enroll subjects with Barth syndrome aged 65 years and older.1
Hepatic impairment is not expected to alter the pharmacokinetics of elamipretide.1
No dosage adjustment is required for patients with mild (eGFR 60 to 89 mL/min) or moderate (eGFR 30 to 59 mL/min) renal impairment.1 The elamipretide dose should be reduced by one half administered subcutaneously once daily in adults with severe renal impairment (eGFR less than 30 mL/min) who are not on dialysis.1 Elamipretide has not been studied in patients with renal failure on dialysis
Most common adverse reactions are injection site reactions.1
It is essential that the manufacturer's labeling be consulted for more detailed information on interactions with this drug, including possible dosage adjustments. Interaction highlights:
Please see product labeling for drug interaction information.1
Elamipretide is a mitochondrial cardiolipin binder that localizes to the inner mitochondrial membrane and improves mitochondrial morphology and function.1
Additional Information
AHFSfirstRelease™. For additional information until a more detailed monograph is developed and published, the manufacturer's labeling should be consulted. It is essential that the manufacturer's labeling be consulted for more detailed information on usual uses, dosage and administration, cautions, precautions, contraindications, potential drug interactions, laboratory test interferences, and acute toxicity.
Excipients in commercially available drug preparations may have clinically important effects in some individuals; consult specific product labeling for details.
Please refer to the ASHP Drug Shortages Resource Center for information on shortages of one or more of these preparations.
Routes | Dosage Forms | Strengths | Brand Names | Manufacturer |
|---|---|---|---|---|
Parenteral | Injection, for subcutaneous use | 280 mg (of elamipretide) per 3.5 mL (80 mg/mL) | Forzinity® | Stealth BioTherapeutics |
AHFS® Drug Information. © Copyright, 1959-2025, Selected Revisions November 10, 2025. American Society of Health-System Pharmacists, Inc., 4500 East-West Highway, Suite 900, Bethesda, MD 20814.