FDA-approved medicines exist for specific indications

SS-31

Elamipretide (Forzinity) — Barth accelerated approval and MMPOWER-3 miss both stay in view.

Editorial review:

What evidence says

Evidence snapshot

This drawer marks papers. It is not a plan, a dose, or a source. A paper is a bounded report. It is not a protocol. Grades name the object. They are not a safety rating.

Identity lock. SS-31 (also MTP-131, Bendavia; INN elamipretide) is a mitochondria-targeted tetrapeptide that binds cardiolipin on the inner mitochondrial membrane. PubChem CID 11764719; formula C32H49N9O5 / MW 639.8; CAS 736992-21-5. Clinical status (same breath): in September 2025, elamipretide (Forzinity) received US accelerated approval to improve muscle strength in Barth syndrome (adult and pediatric patients meeting labeled weight criteria) — first disease-specific Barth treatment in that approval narrative (PMID 41335372). MMPOWER-3 Phase 3 in primary mitochondrial myopathy did not meet co-primary efficacy endpoints (6MWT / fatigue) (PMID 37268435). Unlabeled “SS-31 vials” are not the Forzinity labeled object. Other programs (e.g. dry AMD, broader mitochondrial myopathy) remain investigational. This drawer marks papers. It is not use advice.

Bottom line

Barth syndrome has a labeled accelerated-approval object plus TAZPOWER open-label extension and natural-history comparison work. Primary mitochondrial myopathy Phase 3 (MMPOWER-3) was negative on co-primary endpoints. Mechanism and cardiolipin-targeting reviews explain the class but do not invent extra indications. Preclinical neuroprotection / organ-injury papers are not human grades. Keep the Barth approval and the PMM miss visible together.

Papers

Labeled / late clinical — Barth syndrome

PMID 41335372 — Shirley 2026

Tested: regulatory milestone review — elamipretide (Forzinity) first approval / accelerated approval narrative for Barth syndrome muscle-strength indication.

Does not show: approval for primary mitochondrial myopathy, cosmetic “mito support,” or every investigational indication under development.

PMID 38602181 — Thompson 2024

Tested: long-term efficacy and safety of elamipretide in Barth syndrome during the 168-week open-label extension of TAZPOWER.

Does not show: a new double-blind placebo-controlled Phase 3 replacement by itself, or MMPOWER-3 success.

PMID 36056411 — Hornby 2022

Tested: natural-history comparison study supporting efficacy assessment of elamipretide in Barth syndrome relative to historical controls (small treated cohort).

Does not show: a large randomized PMM win, or broad cardiomyopathy approval beyond the Barth program framing.

PMID 34623544 — Sabbah 2022

Tested: narrative on elamipretide for Barth syndrome cardiomyopathy (gradual rebuilding of mitochondrial bioenergetics framing).

Does not show: a new primary RCT; secondary commentary.

Primary mitochondrial myopathy — Phase 3 miss

PMID 37268435 — Karaa 2023

Tested: MMPOWER-3 — pivotal Phase 3 randomized, double-blind, placebo-controlled trial of subcutaneous elamipretide in genetically confirmed primary mitochondrial myopathy (6MWT and fatigue co-primaries).

Does not show: efficacy on those co-primaries (Class I evidence of no improvement at 24 weeks vs placebo); tolerability is not an efficacy win.

PMID 39574155 — Karaa 2024

Tested: genotype-specific post hoc analysis of elamipretide in MMPOWER-3 PMM participants.

Does not show: that post hoc genotype signals reverse the failed primary analysis into a labeled PMM indication.

Mechanism / class reviews

PMID 24117165 — Szeto 2014

Tested: review of first-in-class cardiolipin-protective SS peptides as agents to restore mitochondrial bioenergetics.

Does not show: a completed pivotal approval package by itself.

PMID 35037146 — Obi 2022

Tested: review targeting mitochondrial dysfunction with elamipretide.

Does not show: new primary trial data; secondary synthesis.

PMID 39940712 — Tung 2025

Tested: structure / mechanism / therapeutic-potential review of elamipretide.

Does not show: a new Phase 3 result.

PMID 32554501 — Chavez 2020

Tested: mitochondrial protein-interaction landscape of SS-31 (proteomics / target engagement science).

Does not show: human clinical efficacy for any indication.

What is not established

That accelerated Barth approval equals approval for PMM, aging, or general “mitochondrial optimization.” That MMPOWER-3 failed primaries can be ignored. Interchangeability of unlabeled “SS-31 vials” with Forzinity trial/approved material. Preclinical organ-injury models as human treatment claims. This drawer does not provide use advice, schedules, or sourcing.

Selected sources

41335372 38602181 36056411 34623544 37268435 39574155 24117165 35037146 39940712 32554501

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