PP / MONOGRAPH 087 Mitochondria-targeting tetrapeptide

SS-31 / elamipretide

Discovery history, preclinical evidence, human studies, current FDA context, and the questions that still need to be answered.

Evidence is not equivalence.

Research on a named molecule does not establish that a catalog material is the studied drug product. Identity, sequence, salt, formulation, purity, sterility, route, labeling, and indication can change the scientific and regulatory conclusion.

ORIGIN / HISTORY

When was SS-31 / elamipretide first described?

SS-31 belongs to the Szeto–Schiller class of cell-permeable, mitochondria-targeting peptides developed through work led by Hazel Szeto and Peter Schiller in the early 2000s. The drug name is elamipretide. [1]

PRECLINICAL EVIDENCE

What do animal and laboratory studies show?

Preclinical pharmacology may exist, but this registry does not treat animal or in-vitro findings as human outcomes. The literature map should be reviewed for the model, species, comparator, endpoint, and replication status. [2]

Translation limitCell and animal findings can generate mechanisms and safety signals, but they do not predict clinical benefit on their own.

CLINICAL EVIDENCE

Are there human studies?

FDA-approved Sept 2025 (“Forzinity,” accelerated approval) for Barth syndrome; also studied in mito disease, heart failure, dry AMD (mixed). These findings apply only to the studied drug product, formulation, population, route, dose, and indication. [2][4]

Check the live study registry ↗

U.S. REGULATORY STATUS

Is SS-31 / elamipretide FDA cleared or approved?

Drug terminology: FDA generally approves drug products; “clearance” is typically a medical-device pathway.

FDA granted accelerated approval to Forzinity (elamipretide) injection on September 19, 2025, for Barth syndrome in patients weighing at least 30 kg. That approval is product-, formulation-, indication-, and labeling-specific; it does not extend to a catalog material merely labeled SS-31 or elamipretide. [5][6][7]

RESEARCH OUTLOOK

Where is the scientific promise?

The credible research opportunity for SS-31 / elamipretide is to define which findings reproduce across adequately powered trials, which endpoints are clinically meaningful, and how benefit–risk changes by indication and formulation. Existing human evidence cannot establish equivalence to an unverified catalog material. [2][4]

LIMITATIONS / PRECAUTIONS

What should professionals keep in view?

Injection reactions; approved only for a rare disease — other uses off-label/unproven; gray-market vials are not the approved product. [2]

Professional-use boundary

This page does not provide a diagnosis, treatment recommendation, protocol, dose, reconstitution instruction, route, or administration schedule. Any lawful program requires independent regulatory, quality, toxicology, and clinical review.

SOURCE REGISTER

References and live evidence maps

Primary papers support specific historical claims where available. PubMed and ClinicalTrials.gov links are live evidence maps, not proof that every result applies to the exact catalog material. FDA links should be rechecked because regulatory pages and product labels can change.