Elamipretide

FORZINITY is a mitochondrial cardiolipin binder that localizes to the inner mitochondrial membrane and improves mitochondrial morphology and function.

• FORZINITY is indicated to improve muscle strength in adult and pediatric patients with Barth syndrome weighing at least 30 kg. This indication is approved under accelerated approval based on an improvement in knee extensor muscle strength, an intermediate clinical endpoint [see Clinical Studies (14) ] .

• Serious hypersensitivity to elamipretide or any of the excipients in FORZINITY [see Warnings and Precautions (5.2) ].contraindicated

For patients weighing 30 kg and greater, the recommended dosage is 40 mg subcutaneously once daily. ( 2.1 ) Reduce the dose in adults with severe renal impairment. ( 2.2 , 8.6 ). 2.1 Recommended Dosage The recommended dosage of FORZINITY in patients weighing at least 30 kg is 40 mg subcutaneously once daily. Patients should receive the dose at the same time each day. Missed Dose If a dose is missed, skip the dose and take the next dose of FORZINITY at the scheduled time. Do not take a double dose of FORZINITY. 2.2 Dosage Modifications For Renal Impairment Refer to Table 1 for dosage modifications in adults with renal impairment. Table 1: Recommended Dosage in Adults with Renal Impairment Estimated Glomerular Filtration Rate (eGFR) (mL/minute) Recommended Dosage Greater than or equal to 30 mL/minute 40 mg subcutaneously once daily Less than 30 mL/minute and NOT on dialysis 20 mg subcutaneously once daily There is insufficient information to recommend a dosage regimen in adults with eGFR less than 30 mL/minute and on dialysis. There is insufficient information to recommend a dosage regimen in pediatric patients weighing 30 kg or greater with renal impairment. 2.3 Important Administration Instructions FORZINITY is a clear, colorless to yellow aqueous solution that contains the preservative, benzyl alcohol. Visually inspect each vial of FORZINITY for particulate matter and cloudiness prior to administration, whenever solution and container permit. Do not use if the solution is cloudy or particulate matter is present.

Benzyl alcohol toxicity : Do not use in neonates. ( 5.1 ) 5.1 Risk of Benzyl Alcohol Toxicity in Neonates FORZINITY is not approved for use in neonates. 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 2,500 grams) and preterm neonates (gestational age less than 34 weeks) who received benzyl alcohol (BA)-containing drugs intravenously. FORZINITY is not approved for intravenous use [see Dosage and Administration (2.1) ] . 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. The minimum amount of BA at which these serious adverse reactions, including fatal reactions, may occur is not known (FORZINITY contains 20 mg of BA per mL). [See Use in Specific Populations (8.4) .] 5.2 Hypersensitivity Hypersensitivity reactions, including serious allergic reactions requiring emergency medical intervention, have been reported in patients receiving FORZINITY. These reactions have included skin manifestations such as rash, papular lesions, and eczematous dermatitis, as well as respiratory symptoms including cough [see Adverse Reactions (6.1) ] . Reactions may occur within minutes to months after treatment initiation. Monitor patients for signs and symptoms of hypersensitivity reactions during treatment.

Most common adverse reactions are injection site reactions. ( 6.1 ) To report SUSPECTED ADVERSE REACTIONS, contact Stealth BioTherapeutics Inc. at 1-844-444-6486 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch. 6.1 Clinical Trials Experience Because clinical trials are conducted under widely varying conditions, adverse reaction rates observed in the clinical trials of a drug cannot be directly compared to rates in the clinical trials of another drug and may not reflect the rates observed in practice. In the FORZINITY clinical development program, 12 male patients aged 12 to 35 years with genetically-confirmed Barth syndrome received treatment with daily subcutaneous injections of 40 mg FORZINITY. Eleven of these 12 patients were Caucasian. Patients first participated in a double-blind, placebo-controlled crossover trial where they were randomized to one of two sequences: 12 weeks of FORZINITY in Period 1 then a 4-week washout followed by 12 weeks of placebo in Period 2 or 12 weeks of placebo in Period 1 then a 4-week washout followed by 12 weeks of FORZINITY in Period 2 Ten patients completed the randomized trial and entered the open-label extension period where they received FORZINITY once daily. Eight of these patients received FORZINITY for 168 weeks, three of whom received FORZINITY for a total of 192 weeks. Adverse reactions occurring more commonly on FORZINITY than on placebo include injection site reactions such as injection site erythema, pain, induration, pruritus, bruising, and urticaria (Table 2).

8.1 Pregnancy Risk Summary Barth Syndrome is a rare, X-linked, recessive, genetic disorder and is not likely to affect females. Therefore, there are no data with FORZINITY use in pregnant women to evaluate for a drug-related risk of major birth defects, miscarriage, or other adverse maternal or fetal outcomes. In animal reproduction studies, no adverse developmental outcomes occurred at any dose tested (see Data ). FORZINITY contains benzyl alcohol as a preservative. Because benzyl alcohol is rapidly metabolized by a pregnant woman, benzyl alcohol exposure in the fetus is unlikely. The background risk of major birth defects and miscarriage for the indicated population is unknown. All pregnancies have a background risk of birth defect, loss, or other adverse outcomes. In the U.S. 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. Data Animal Data In pregnant rats, once-daily intravenous infusion of elamipretide during the period of organogenesis [gestation day (GD) 7 to GD 17] did not result in embryo-fetal developmental toxicity at doses tested up to 10 mg/kg/day, approximately 4 times the clinical exposure at the maximum recommended human dose (MRHD) of 40 mg/day, based on area under the concentration-time curve (AUC).

Metabolism

Elamipretide exposure increases proportionally over a dose range of 2 to 80 mg following daily subcutaneous injections with minimal accumulation. Absorption Maximum elamipretide concentrations were reached between 0.5 to 1 hour after subcutaneous administration.

There were no reports of overdose during clinical trials with FORZINITY. Symptoms and signs of overdose are likely to be histamine-related (e.g., decreased blood pressure, presyncope) and should be managed according to standard of care. Interrupt treatment with FORZINITY if there is suspicion of overdose.

• Sep 19, 2025NDANDA215244Stealth Biotheraps

The 10 most recently updated of 21 ClinicalTrials.gov registrations naming Elamipretide as an intervention. Registration is not evidence of efficacy or safety — reference crosswalk only.

• ReNEW:Phase 3 Study of Efficacy, Safety & Pharmacokinetics of Subcutaneous Injections of Elamipretide in Subjects With Dry Age-Related Macular Degeneration (Dry AMD)
  Active not recruiting · Phase 3 · Interventional · 313 enrolled · Stealth BioTherapeutics Inc.
  NCT06373731updated 2026-05-20
• Clinical Trial in Patients With Barth Syndrome- 4TAZPower
  Not yet recruiting · Phase 4 · Interventional · 48 enrolled · Stealth BioTherapeutics Inc.
  NCT07531251updated 2026-05-06
• FRDA Investigator Initiated Study (IIS) With Elamipretide
  Completed · Phase 1 · Phase 2 · Interventional · 20 enrolled · Children's Hospital of Philadelphia
  NCT05168774updated 2025-12-12
• Study of Healthy Aging and Physical Function With Elamipretide
  Recruiting · Phase 2 · Interventional · 30 enrolled · David Marcinek
  NCT07275424updated 2025-12-10
• Study to Evaluate Efficacy and Safety of Elamipretide in Subjects With Primary Mitochondrial Disease From Nuclear DNA Mutations (nPMD)
  Completed · Phase 3 · Interventional · 102 enrolled · Stealth BioTherapeutics Inc.
  NCT05162768updated 2025-10-24
• An Intermediate Size Expanded Access Protocol of Elamipretide
  Available · Expanded access · Stealth BioTherapeutics Inc.
  NCT04689360updated 2025-02-14
• ReCLAIM-2 Study to Evaluate Safety,Efficacy & Pharmacokinetics of Elamipretide in Subjects With AMD With Non-central GA
  Completed · Phase 2 · Interventional · 176 enrolled · Stealth BioTherapeutics Inc.
  NCT03891875updated 2024-07-12
• A Trial to Evaluate Safety, Tolerability and Efficacy of Elamipretide in Subjects With Barth Syndrome
  Completed · Phase 2 · Phase 3 · Interventional · 12 enrolled · Stealth BioTherapeutics Inc.
  NCT03098797updated 2024-04-16
• A Trial to Evaluate Safety and Efficacy of Elamipretide Primary Mitochondrial Myopathy Followed by Open-Label Extension
  Terminated · Phase 3 · Interventional · 218 enrolled · Stealth BioTherapeutics Inc.
  NCT03323749updated 2022-01-24
• Open-Label Extension Trial to Characterize the Long-term Safety and Tolerability of Elamipretide in Subjects With Genetically Confirmed Primary Mitochondrial Myopathy (PMM)
  Terminated · Phase 2 · Interventional · 28 enrolled · Stealth BioTherapeutics Inc.
  NCT02976038updated 2021-12-17

Frequently asked questions

How does Elamipretide work?

FORZINITY is a mitochondrial cardiolipin binder that localizes to the inner mitochondrial membrane and improves mitochondrial morphology and function.

What is Elamipretide used for?

According to FDA labeling, Elamipretide carries indications including: FORZINITY is indicated to improve muscle strength in adult and pediatric patients with Barth syndrome weighing at least 30 kg. This indication is approved under accelerated approval based on an improvement in knee extensor muscle strength, an intermediate clinical endpoint [see Clinical Studies (14) ] .. This is a reference summary of labeled uses, not medical advice or a treatment recommendation.

What class of drug is Elamipretide?

Elamipretide is classified as Increased Metabolic Rate.

What are the brand names for Elamipretide?

Elamipretide is marketed under brand names including Forzinity.

What are the contraindications for Elamipretide?

Elamipretide labeling lists contraindications including: Serious hypersensitivity to elamipretide or any of the excipients in FORZINITY [see Warnings and Precautions (5.2) ].. Always consult the full prescribing information and a clinician.