Elamipretide

Definition

Elamipretide is a synthetic tetrapeptide that concentrates in the inner mitochondrial membrane and binds cardiolipin, the phospholipid whose remodeling fails in Barth syndrome; the label gives its sequence as D-Arg-2,6-dimethyl-Tyr-Lys-Phe-NH2 and calls the product a mitochondrial cardiolipin binder ^[1].

It reaches this index through the company it keeps rather than through any market of its own. Providers who sell NAD+ and list MOTS-c as a mitochondrial peptide are describing a category whose only approved member is this one, and that member's label names a single rare genetic disorder.

Evidence

Across five name variants PubMed returns 470 indexed records, of which 201 carry the Humans subject heading, 22 the clinical-trial publication type and 19 the randomized-controlled-trial type ^[2]. All twenty-two were opened and read; eighteen name a variant, four are matches on the search rather than on the compound, and one of the eighteen is a rat study that carries a trial publication type anyway ^[2].

The approved indication rests on a program of about a dozen participants. TAZPOWER randomized twelve subjects to elamipretide or placebo in a twelve-week crossover and neither primary endpoint was met; ten continued into an open-label extension, where the six-minute walk distance improved by 95.9 meters at 36 weeks at a p value of 0.024 ^[3]. The 168-week extension reported sustained tolerability and a cumulative 96.1 meter improvement at week 168, at a p value of 0.003, in the eight patients who reached that visit ^[4].

The largest controlled trial of the compound failed, and it failed in a different disease. MMPOWER-3 randomized 218 adults with genetically confirmed primary mitochondrial myopathy and met neither primary endpoint, with a six-minute walk difference of minus 3.2 meters at a p value of 0.69 ^[5].

Two further randomized trials sit outside the approved indication and neither produced one: a placebo-controlled trial in heart failure ^[6], and a trial of a topical ophthalmic solution in Leber hereditary optic neuropathy, which is not even the approved route ^[7]. A reader meeting the word mitochondrial on a provider menu is meeting a compound whose record is a rare-disease program with one positive extension and one negative phase 3.

Side effects

The controlled safety data behind the Forzinity label are small: 12 males aged 12 to 35 with Barth syndrome, in a placebo-controlled crossover trial followed by an open-label extension ^[11]. Injection site reactions were the adverse reactions seen more often on the drug than on placebo. Every one of the 12 had injection site redness on elamipretide against 3 on placebo, and pain, hardening, itching, bruising and hives at the injection site were also more common on the drug ^[11].

Blood eosinophil counts rose frequently in studies lasting 30 days or longer. The label states that the counts generally peaked around 90 days, returned to baseline with continued treatment or after stopping, and were not linked to symptoms or other laboratory changes ^[11].

Hypersensitivity is the main warning. Reactions, some requiring emergency treatment, have included rash, raised skin lesions, eczema-like dermatitis and cough, and they may begin within minutes or months after starting treatment; the drug is contraindicated after a serious hypersensitivity reaction to elamipretide or its ingredients ^[11].

The product contains benzyl alcohol as a preservative, and the label warns that benzyl alcohol has caused serious and fatal reactions in premature and low-birth-weight newborns; Forzinity is not approved for newborns or for intravenous use ^[11]. Barth syndrome rarely affects females, so there are no data on use in pregnancy ^[11].

Educational summary of the cited sources, not medical advice; a prescriber weighs these risks against a person’s own history.

Lawful access

The lawful supply route is the approved product. FDA approved elamipretide injection under new drug application 215244 on September 19, 2025 as a Type 1 new molecular entity, and Drugs@FDA records Forzinity as a subcutaneous solution of elamipretide hydrochloride at 280 mg base per 3.5 mL, sponsored by Stealth BioTherapeutics and marketed as prescription-only ^[8].

The label states the indication and the condition attached to it in the same breath. In the version effective December 10, 2025 the product is indicated "to improve muscle strength in adult and pediatric patients with Barth syndrome weighing at least 30 kg", and that indication "is approved under accelerated approval based on an improvement in knee extensor muscle strength, an intermediate clinical endpoint", with continued approval that "may be contingent upon verification and description of clinical benefit in a confirmatory trial" ^[1].

Compounding does not arise for this molecule. Elamipretide appears in none of the three categories of the section 503A bulks list published May 14, 2026 ^[9], and FDA's drug shortage database holds no entry for it ^[10].

Related molecules

Elamipretide is filed with the mitochondrial group beside NAD+, glutathione and MOTS-c, and the four together answer the question that group exists to answer. One has an approved product with a rare-disease label, two are compounded under an interim listing while FDA evaluates them, and one may not be compounded at all.

The approved peptide drugs table places this product beside the rest of the approved set, and the human trial record carries the census behind the counts above. The compoundable page lists what is lawfully obtainable in this group today, which is not this molecule.

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