Elamipretide (SS-31)

Classification

Mitochondria-targeted tetrapeptide (investigational)

Approval status

Investigational · Not approved for human use

Research status

Investigational (elamipretide). Not broadly approved as a mitochondrial medicine at last review; development status should be re-checked against FDA/EMA/TGA registers.

Molecular formula

Insufficient reliable evidence identified.

Molecular weight

Insufficient reliable evidence identified.

Sequence

D-Arg-Dmt-Lys-Phe-NH2 (aromatic-cationic tetrapeptide)

Primary biological target

Proposed: cardiolipin on the inner mitochondrial membrane

Half-life

Short plasma half-life with intracellular mitochondrial targeting described in development literature

Routes investigated

Intravenous, Subcutaneous (development programmes), Topical ophthalmic (separate programme)

Human evidence level

C — Limited or early human evidence

Investigational mitochondria-targeted peptide. Not sold.

Synthetic mitochondria-targeted tetrapeptide (SS-peptide class).

Proposed mechanism

Proposed mechanism: elamipretide associates with cardiolipin, aiming to improve inner-membrane structure and electron-transport efficiency in stressed mitochondria. Mechanism not yet fully established as an approved systemic indication. Clinical programmes in primary mitochondrial disease and Barth syndrome have been mixed or indication-specific — check current regulatory status rather than assuming approval.

Not an approved Australian mitochondrial medicine.

• Mitochondrial disease programmes

  C · Limited or early human evidence

  Early human research has examined elamipretide in primary mitochondrial myopathy and related conditions; results and regulatory outcomes are programme-specific.^[1]

Investigational findings are not proven clinical benefits unless an authority has labelled that indication.

Overall human evidence grade: C · Limited or early human evidence

Animal, cell and in-vitro findings are listed separately from human effects.

Extensive mitochondrial-model literature.

This section does not provide personalised medical dosing. Values are those reported in papers or protocols. Animal mg/kg doses are not converted to human doses.

Doses reported in human studies

1. Elamipretide mitochondrial myopathy development (representative)

   Dose: Protocol-specific (see MMPOWER papers) mg

   Route: Subcutaneous (typical development route)

   Frequency: Once daily in several protocols

   Duration: Study-specific

   Population: Primary mitochondrial myopathy

   Purpose: Mitochondrial myopathy efficacy/safety

   ^[1]

Preclinical / animal study dosing

Insufficient reliable evidence identified.

Development PK exists in papers and investigator brochures; not a consumer PI.

Injection-site reactions have been reported in development.

Unknowns. Long-term unapproved use is not characterised.

Contraindications. Not for unapproved use.

Australia — TGA

Not an established ARTG mitochondrial peptide for general use — verify register.

United States — FDA

Development/regulatory status is indication-specific; not a general wellness approval.

European Union — EMA

Verify register; do not assume approval.

1. [1] Randomized dose-escalation trial of elamipretide in adults with primary mitochondrial myopathy

   Karaa A et al. Randomized dose-escalation trial of elamipretide in adults with primary mitochondrial myopathy Neurology. 2018. doi:10.1212/WNL.0000000000005255 PMID 29626192

   PMID 29626192DOI