Elamipretide
Elamipretide is studied as a mitochondria-targeted tetrapeptide, described in fda labelling as a mitochondrial cardiolipin binder in biochemical and physiological models.
Elamipretide Biological Classification
Investigated within physiological and chemical models.
What kind of compound
Peptide
Mitochondria-targeted tetrapeptide, described in FDA labelling as a mitochondrial cardiolipin binder.
Human testing on file
0human records
0 animal · 0 total indexed. No plain-English evidence summary on file.
Regulatory position
FDA-approved drug substance
Studied for
Mitochondria-targeted peptides
Reviewed 15 September 2026
Known safety concerns in this file
- No sourced human safety summary is recorded for this compound.
Commonly compared with
No comparison set is recorded for this compound yet.
Open side-by-side comparison →What is Elamipretide?
Elamipretide is classified as a mitochondria-targeted tetrapeptide, described in fda labelling as a mitochondrial cardiolipin binder. An approved mitochondria-targeted tetrapeptide, marketed as FORZINITY for Barth syndrome under FDA's accelerated approval pathway.
Why are people interested in it?
Researchers and clinicians study Elamipretide primarily to understand its biological interactions, receptor binding affinity, and potential role within its biochemical class.
Primary Research Categories
- Mitochondria-targeted peptides
- Approved drug substances
How does it work in the body?
Elamipretide interacts with specific cellular receptors and metabolic pathways corresponding to its structural classification as a mitochondria-targeted tetrapeptide, described in fda labelling as a mitochondrial cardiolipin binder.
Biological Pathway Overview
Elamipretide Receptor & Target Interactions
This compound is evaluated in scientific research for its interactions with target cellular receptors and signaling cascades. Consult the evidence section below for specific peer-reviewed papers.
Target Pathway
Engages specific cellular signaling cascades characteristic of its structural family.
Endocrine/Paracrine Activity
Investigated for its regulatory effects on local tissue synthesis and cellular signaling.
What has actually been demonstrated in humans?
Evidence for Elamipretide is currently derived primarily from laboratory or preclinical models. Consult the published evidence file for specific indexed studies.
Pharmaceutical Reality vs. Unregulated Research Chemicals
Elamipretide is not an FDA-approved medication. Always verify chemical identity and testing documentation before interpreting research literature.
What is still uncertain or unknown?
Controlled human clinical trials evaluating Elamipretide for therapeutic efficacy, safety, and pharmacokinetics are limited or absent in peer-reviewed Western registries.
Companion Resources & Guides
Research at a Glance
Open the full evidence file (0 records)No evidence records in this file yet.
What Has Actually Been Studied
What is Still Unknown
What we know
Editorial reading of the records below, labelled as Therapept analysis. How records are built
What remains uncertain
Preparation and Handling
Lyophilized peptides ship as freeze-dried powders; whatever happens next involves diluents, concentrations and storage conditions that are compound- and product-specific.
United States Regulatory Status
FDA-approved drug substance
Application: NDA 215244 (FORZINITY)
Approved Indication: Marketed in the United States as FORZINITY, approved under accelerated approval to improve muscle strength in adult and paediatric patients with Barth syndrome above a label-specified body weight. Continued approval may be contingent on verification of clinical benefit in a confirmatory trial.
- FORZINITY (elamipretide hydrochloride), NDA 215244, approved 19 September 2025 as a new molecular entity under priority review.
- The indication is approved under accelerated approval based on improvement in knee extensor muscle strength, an intermediate clinical endpoint.
- Not named on the WADA 2026 Prohibited List.
Technical Chemical SpecificationsClick to view CAS, sequence, and molecular formulas
- Molecular weight
- 639.8 g/mol as the free base (PubChem); 749.2 as the trihydrochloride (FDA label)
- Sequence length
- 34 chars
- CAS number
- 736992-21-5
- PubChem CID
- 11764719
- Compound class
- Mitochondria-targeted tetrapeptide, described in FDA labelling as a mitochondrial cardiolipin binder
- Sequence
- D-Arg-2,6-dimethyl-Tyr-Lys-Phe-NH2
- Molecular formula
- C32H49N9O5
- Molecular weight
- 639.8 g/mol as the free base (PubChem); 749.2 as the trihydrochloride (FDA label)
- CAS number
- 736992-21-5
- PubChem CID
- 11764719
Sources
Last reviewed 15 September 2026 · Compiled by Therapept Editorial · How these records are built