Elamipretide (SS-31)

Elamipretide is a real, regulated drug with clinical trial data behind its approved use, available only by prescription, for a specific ultra-rare disease. This page describes that approved use — that's public medical information about a regulated drug, not personalized medical advice, and not a basis for off-label, general-population use. Talk to a doctor about whether it's appropriate for you.

Elamipretide (also known by its research name SS-31) is a small, mitochondria-targeted peptide. Unlike the GH-axis or GLP-1 peptides elsewhere in this section, its proposed mechanism is to bind cardiolipin, a lipid in the inner mitochondrial membrane, helping stabilize the membrane structure that mitochondria need to produce cellular energy efficiently. This mechanism is why it's being studied across a range of diseases where mitochondrial dysfunction plays a central role.

The FDA granted accelerated approval to elamipretide HCl, under the brand name Forzinity, on September 19, 2025 — the first approved treatment for Barth syndrome, an ultra-rare, life-limiting genetic mitochondrial disease affecting an estimated 150 people in the US, primarily boys and men, causing cardiac and skeletal muscle complications. It's approved for adult and pediatric patients weighing at least 30 kg. Approval was based on the TAZPOWER trial, using improved knee extensor muscle strength as an intermediate clinical endpoint over 168 weeks, and came with Orphan Drug, Fast Track, Priority Review, and Rare Pediatric Disease designations. Because this was an accelerated approval, continued approval may depend on confirmatory trials verifying clinical benefit.

Barth Syndrome Foundation — FDA Approves First Medication for Barth Syndrome →

Elamipretide's approval is narrow — Barth syndrome specifically — but its mitochondria-targeted mechanism has driven trials in other conditions where mitochondrial function is implicated, including geographic atrophy (an eye disease), heart failure with preserved ejection fraction, and broader interest in aging-related mitochondrial decline. None of that broader research constitutes approval for those uses — it's ongoing investigational work, and using elamipretide for any purpose outside its approved indication would be off-label and unstudied for that purpose.

As with any newly approved therapy, the full long-term safety profile continues to be characterized post-approval. Reported effects in trials have generally centered on injection-site reactions, consistent with a subcutaneously administered peptide. Given the rarity of Barth syndrome and the small trial population behind its approval, safety monitoring in real-world use is ongoing.

Pharmacy Times — FDA Grants Accelerated Approval to Elamipretide