U.S. approval recordRare disease

Elamipretide

Related names in cited records: SS-31, MTP-131, Forzinity

FDA granted Forzinity (elamipretide) accelerated approval to improve muscle strength in adults and children with Barth syndrome weighing at least 30 kg.

Citation markers ↗ open the public records supporting the adjacent statements.

Correction · September 15, 2026

We added a preserved FDA-hosted product label to support the product-specific label account. Prior profile versions retain their original citations. 3

This version supersedes public version 3.

Start here

The evidence at a glance

What is its status in the cited records?

U.S. accelerated approval for a specified Barth syndrome population. FDA's September 19, 2025 approval letter requires a confirmatory trial to verify clinical benefit. The approval concerns this product and specified use; a recruiting trial record does not describe its full regulatory status.

Status sources checked through September 15, 2026. See status sources and coverage →

What do the reviewed human studies say?

Human evidence reviewed September 15, 2026 · Review version 1.

Review source snapshots checked September 15, 2026 · Integrity checked September 15, 2026.

Three selected reports cover the randomized Barth syndrome study, follow-up of the same cohort, and a separate primary mitochondrial myopathy trial. They do not cover every indication or replace the product-specific regulatory record.

What the reviewed sources report

The randomized Barth syndrome study did not meet its two primary endpoints; later uncontrolled follow-up reported improvements. The larger MMPOWER-3 trial in primary mitochondrial myopathy also missed its primary endpoints. These populations and study designs must be considered separately.

What remains unresolved?

  • Which confirmatory results establish clinical benefit in the Barth syndrome population?
  • How do study population, attrition and the absence of a control group affect the extension findings?

Read study designs, results, limitations, and sources →

What does the U.S. product label say?

FORZINITY, revised September 2025 · Read September 15, 2026 · Preserved label summary 1

Which population and use does the label cover?

The label covers improved muscle strength in adults and children with Barth syndrome weighing at least 30 kg. Accelerated approval rests on an intermediate clinical endpoint and requires confirmation of clinical benefit. Label: 1 ↗

Which safety limits matter?

The label contraindicates serious hypersensitivity and warns about hypersensitivity reactions and benzyl-alcohol toxicity in neonates. Injection-site reactions were the most common adverse reactions. Label: 4, 5, 6 ↗

Label edition and source pins

FDA-hosted U.S. prescribing information. This account concerns this product and label edition; other formulations and jurisdictions require their own records.

FORZINITY, revised September 2025: U.S. prescribing information

Label PDF SHA-256: 1a32a3b7452b7c33c4a60b7f9175f80fcce499775feb39b4c14743293e6bf618.

Open versioned label context data →

Reviews summarize selected studies; their scope and limitations are shown above.

Inside the official reviews

Reviewed 2026-09-19 · Preserved version 1

The randomized trial did not meet its primary endpoints

FDA’s 2025 review reports that the 12-participant, placebo-controlled crossover study did not show superiority on six-minute walk distance or fatigue. The two 12-week treatment periods and later open-label follow-up answer different questions. FDA PDF pp. 3, 85 ↗

More findings, study links and document coverage

Why the accelerated-approval decision differed

The signatory accepted sustained knee-extension strength findings despite the review team’s recommendation against approval. The decision acknowledged substantial uncertainty from unblinded assessment, attrition and the absence of a concurrent control in the extension. FDA PDF pp. 3, 4 ↗

Muscle strength was an intermediate clinical endpoint

FDA distinguished the knee-extensor measurement from demonstrated improvement in how patients function. Accelerated approval used knee-extensor strength as an intermediate clinical endpoint and required a trial to confirm clinical benefit; this document does not establish that trial’s subsequent outcome. FDA PDF pp. 4 ↗

The scope is Barth syndrome

This assessment concerns Forzinity in Barth syndrome. Neither its decision nor the extension findings establish efficacy for unrelated mitochondrial conditions or general performance enhancement. FDA PDF pp. 2, 3, 4 ↗

Read the separate U.S. label account → Label version and indication scope are preserved there; these agency reviews describe their own dated evidence.

Matched study identities

Protocol identifiers were matched to ClinicalTrials.gov records. This does not count extensions as new independent trials.

Document coverage

Selected sections read in 1 of 1 inventoried documents; 0 not yet reviewed. Reading selected sections does not establish complete appraisal of a document.

Selected English EMA PDFs from the retained September 15, 2026 title-matched document feed, plus independently retrieved FDA approval-package documents where listed. Candidate entries are not verified product/application matches. This is a bounded inventory, not a complete FDA, national or worldwide source search. EMA inventory source ↗

Inspect documents, application identities and source pins

Page references use one-based PDF file pages, which can differ from printed page numbers. Candidate feed associations are not verified application identities.

  • Forzinity: FDA 2025 integrated review ↗

    Selected sections reviewed · fda-forzinity-integrated-2025 · Document/feed date: 2025-09-19

    Independently retrieved FDA approval-package PDF. The displayed date belongs to the selected review, not the entire compilation or a current label.

    Verified: Forzinity · NDA 215244 · Document dated 2025-09-19. Identity checked on PDF pp. 1, 2.

    Identity and selected clinical design, efficacy, safety and benefit-risk sections only. One-based PDF file pages; unlisted pages have not been appraised. The date identifies the selected review within any compiled approval package.

    Retrieved 2026-09-19. Read PDF pages: 1, 2, 3, 4, 85 of 378. SHA-256: 354af3d2efb096379db185bcbd181635c91bbe0c1a3a2327c375a16f820f2415.

Remaining work

  • Read the full integrated efficacy and safety sections, and reconcile the confirmatory-trial record and later regulatory updates.
  • Keep other mitochondrial-disease trials separate from the Barth syndrome evidence.

Open versioned document coverage and study pins →

Research coverage and study families

Search and screening record checked 2026-09-15. Counts describe records, not independent trials. Full-text appraisal is separate from an abstract review.

Open machine-readable coverage and record decisions →

PubMed search

Primary reports of administration to people of the specified peptide; secondary reviews, animal/cell experiments and unrelated acronym matches are outside this scope. Selected summaries do not close the broader screening queue.

447 retrieved · 3 included · 0 excluded · 444 awaiting a decision · 2 full-text appraisals

Search terms, record decisions and remaining work

"elamipretide"[Title/Abstract] OR "SS-31"[Title/Abstract] OR "MTP-131"[Title/Abstract] OR "Forzinity"[Title/Abstract]

  • Search covers PubMed title/abstract terms; regulator, registry, older aliases, non-indexed and non-English records require separate reconciliation.
  • Full-text appraisal and independent registry comparison remain incomplete.

Showing 25 of 447 record decisions, with included studies and unresolved records first. The versioned data inventory retains every record and study-family link.

  1. PubMed 38602181 — include: Selected human report; abstract and record metadata reviewed. Study families: NCT03098797.
  2. PubMed 37268435 — include: Selected human report; main full text and current registry comparison reviewed. Specific supplements, historical comparisons and unresolved issues remain identified in the structured reading. Study families: NCT03323749.
    Structured full-text reading · PMC10382259

    This is an editorial structured reading, not a validated risk-of-bias score.

    Read the source full text (Europe PMC XML) ↗ · NCT03323749 registry record ↗

    Randomization and masking
    MMPOWER-3 used computer-generated assignment, genotype stratification and identical vials; participants, investigators, pharmacists and sponsor were blinded. There were 218 randomized participants, 109 per group.
    Missing data
    The paper reports 205 completing 24 weeks and uses mixed-model repeated measures in the treated population. Text and figure disagree on per-protocol counts; that discrepancy remains unresolved. Adverse-event discontinuations were eight versus two, a different measure from completion.
    Outcome selection
    Neither coprimary endpoint improved: the between-group six-minute walk difference was -3.2 m (95% CI -18.7 to 12.3), and fatigue also missed significance. A post hoc genotype subgroup signal does not reverse the primary result. The paper reports five versus three serious adverse events, attributed as unrelated by investigators.
    Applicability
    Participants had ambulatory primary mitochondrial myopathy; 94% were White and 64% female. Eligibility, baseline walking ability and mostly mild-to-moderate fatigue restrict generalization. This is a different population from the Barth syndrome indication.
    Funding and conflicts
    Stealth BioTherapeutics funded and helped design the study. Sponsor staff and compensated consultants were among the authors; the lead author had full data access and others received aggregate data.
    Registry comparison
    The retained NCT03323749 record agrees on the two 24-week primary outcomes and reports termination after primary endpoint failure. Secondary-outcome history and linked supplements have not been fully reconciled. The current record is not a complete historical protocol audit.

    Reviewed 2026-09-15; source SHA-256 35fbf3139ae775f5ff4d3a011ce2461ea5cf7dc8eef296f1f8abf47cc56f1342.

  3. PubMed 33077895 — include: Selected human report; main full text and current registry comparison reviewed. Specific supplements, historical comparisons and unresolved issues remain identified in the structured reading. Study families: NCT03098797.
    Structured full-text reading · PMC7935714

    This is an editorial structured reading, not a validated risk-of-bias score.

    Read the source full text (Europe PMC XML) ↗ · NCT03098797 registry record ↗

    Randomization and masking
    SPIBA-201 used independent randomization and identical packaging in a blinded crossover: two 12-week periods separated by washout. All 12 participants completed both randomized periods.
    Missing data
    Ten entered the uncontrolled extension; two stopped after injection-site reactions and eight contributed week-36 outcomes. Extension improvements therefore describe a selected continuing group, not all randomized participants.
    Outcome selection
    The randomized walking and fatigue coprimary outcomes were null; the walking difference was -0.8 m (p=0.97). Extension efficacy was secondary and based on within-person changes. One randomized-period serious event, hospitalization for costochondritis, was reported as unrelated; absence of serious harm is not supported.
    Applicability
    This single-site study enrolled ambulatory males aged at least 12 with genetically confirmed Barth syndrome. It cannot establish effects in infants or all disease severities. Uncontrolled extension results remain vulnerable to learning effects and other changes over time.
    Funding and conflicts
    Stealth BioTherapeutics funded the study and contributed to design, analysis and writing, but not data collection, according to the authors.
    Registry comparison
    NCT03098797 matches the 12-participant crossover and randomized walking/fatigue outcomes. The 168-week publication follows the same study family. Supplementary outcome definitions and the complete registry history remain unreviewed; extension exposure duration must not be confused with calendar follow-up.

    Reviewed 2026-09-15; source SHA-256 ea8b01a5464860686aba02cae7e90c580dc554558f0b00b68f4745f33b106277.

  4. PubMed 42357949 — not screened: Retrieved for the broader search; eligibility has not yet been adjudicated.
  5. PubMed 42600764 — not screened: Retrieved for the broader search; eligibility has not yet been adjudicated.
  6. PubMed 42473606 — not screened: Retrieved for the broader search; eligibility has not yet been adjudicated.
  7. PubMed 42709510 — not screened: Retrieved for the broader search; eligibility has not yet been adjudicated.
  8. PubMed 42448476 — not screened: Retrieved for the broader search; eligibility has not yet been adjudicated.
  9. PubMed 42324025 — not screened: Retrieved for the broader search; eligibility has not yet been adjudicated.
  10. PubMed 42242082 — not screened: Retrieved for the broader search; eligibility has not yet been adjudicated.
  11. PubMed 42708867 — not screened: Retrieved for the broader search; eligibility has not yet been adjudicated.
  12. PubMed 42638278 — not screened: Retrieved for the broader search; eligibility has not yet been adjudicated.
  13. PubMed 42676996 — not screened: Retrieved for the broader search; eligibility has not yet been adjudicated.
  14. PubMed 42570043 — not screened: Retrieved for the broader search; eligibility has not yet been adjudicated.
  15. PubMed 42546889 — not screened: Retrieved for the broader search; eligibility has not yet been adjudicated.
  16. PubMed 42361626 — not screened: Retrieved for the broader search; eligibility has not yet been adjudicated.
  17. PubMed 42290373 — not screened: Retrieved for the broader search; eligibility has not yet been adjudicated.
  18. PubMed 41966639 — not screened: Retrieved for the broader search; eligibility has not yet been adjudicated.
  19. PubMed 41934921 — not screened: Retrieved for the broader search; eligibility has not yet been adjudicated.
  20. PubMed 41233677 — not screened: Retrieved for the broader search; eligibility has not yet been adjudicated.
  21. PubMed 42456009 — not screened: Retrieved for the broader search; eligibility has not yet been adjudicated.
  22. PubMed 42443448 — not screened: Retrieved for the broader search; eligibility has not yet been adjudicated.
  23. PubMed 42358073 — not screened: Retrieved for the broader search; eligibility has not yet been adjudicated.
  24. PubMed 42415111 — not screened: Retrieved for the broader search; eligibility has not yet been adjudicated.
  25. PubMed 42359884 — not screened: Retrieved for the broader search; eligibility has not yet been adjudicated.

ClinicalTrials.gov search

ClinicalTrials.gov API v2 identity-name search, complete retrieved records. Includes protocols, extensions, combinations and expanded access; these are not all efficacy trials or published outcomes.

32 retrieved · 30 included · 2 excluded · 0 awaiting a decision · 0 full-text appraisals

Search terms, record decisions and remaining work

elamipretide OR MTP-131 OR SS-31 OR bendavia

Source retrievals and payload pins
  • Open the source endpoint ↗

    Retrieved 2026-09-15T08:28:05.001641+00:00 · HTTP 200 · SHA-256 17a734e9b19b212b69514e8299bf42af0e4f310d7da662e5ecf0dee66b1ea7cb.

  • Registry identity screening is distinct from appraisal of posted results, protocol history and publication linkage.
  • Other registries and unregistered studies are outside this search.

Showing 25 of 32 record decisions, with included studies and unresolved records first. The versioned data inventory retains every record and study-family link.

  1. ClinicalTrials.gov NCT02848313 — include: The intervention record names this molecule or its validated development code. Eligibility here is registry identity matching; outcomes and applicability remain separately unappraised. Study families: NCT02848313.
  2. ClinicalTrials.gov NCT07531251 — include: The intervention record names this molecule or its validated development code. Eligibility here is registry identity matching; outcomes and applicability remain separately unappraised. Study families: NCT07531251.
  3. ClinicalTrials.gov NCT07275424 — include: The intervention record names this molecule or its validated development code. Eligibility here is registry identity matching; outcomes and applicability remain separately unappraised. Study families: NCT07275424.
  4. ClinicalTrials.gov NCT02693119 — include: The intervention record names this molecule or its validated development code. Eligibility here is registry identity matching; outcomes and applicability remain separately unappraised. Study families: NCT02693119.
  5. ClinicalTrials.gov NCT02805790 — include: The intervention record names this molecule or its validated development code. Eligibility here is registry identity matching; outcomes and applicability remain separately unappraised. Study families: NCT02805790.
  6. ClinicalTrials.gov NCT02914665 — include: The intervention record names this molecule or its validated development code. Eligibility here is registry identity matching; outcomes and applicability remain separately unappraised. Study families: NCT02914665.
  7. ClinicalTrials.gov NCT01754818 — include: The intervention record names this molecule or its validated development code. Eligibility here is registry identity matching; outcomes and applicability remain separately unappraised. Study families: NCT01754818.
  8. ClinicalTrials.gov NCT01755858 — include: The intervention record names this molecule or its validated development code. Eligibility here is registry identity matching; outcomes and applicability remain separately unappraised. Study families: NCT01755858.
  9. ClinicalTrials.gov NCT05162768 — include: The intervention record names this molecule or its validated development code. Eligibility here is registry identity matching; outcomes and applicability remain separately unappraised. Study families: NCT05162768.
  10. ClinicalTrials.gov NCT02976038 — include: The intervention record names this molecule or its validated development code. Eligibility here is registry identity matching; outcomes and applicability remain separately unappraised. Study families: NCT02976038.
  11. ClinicalTrials.gov NCT01115920 — include: The intervention record names this molecule or its validated development code. Eligibility here is registry identity matching; outcomes and applicability remain separately unappraised. Study families: NCT01115920.
  12. ClinicalTrials.gov NCT03891875 — include: The intervention record names this molecule or its validated development code. Eligibility here is registry identity matching; outcomes and applicability remain separately unappraised. Study families: NCT03891875.
  13. ClinicalTrials.gov NCT01572909 — include: The intervention record names this molecule or its validated development code. Eligibility here is registry identity matching; outcomes and applicability remain separately unappraised. Study families: NCT01572909.
  14. ClinicalTrials.gov NCT02436447 — include: The intervention record names this molecule or its validated development code. Eligibility here is registry identity matching; outcomes and applicability remain separately unappraised. Study families: NCT02436447.
  15. ClinicalTrials.gov NCT02814097 — include: The intervention record names this molecule or its validated development code. Eligibility here is registry identity matching; outcomes and applicability remain separately unappraised. Study families: NCT02814097.
  16. ClinicalTrials.gov NCT05168774 — include: The intervention record names this molecule or its validated development code. Eligibility here is registry identity matching; outcomes and applicability remain separately unappraised. Study families: NCT05168774.
  17. ClinicalTrials.gov NCT01513200 — include: The intervention record names this molecule or its validated development code. Eligibility here is registry identity matching; outcomes and applicability remain separately unappraised. Study families: NCT01513200.
  18. ClinicalTrials.gov NCT02388464 — include: The intervention record names this molecule or its validated development code. Eligibility here is registry identity matching; outcomes and applicability remain separately unappraised. Study families: NCT02388464.
  19. ClinicalTrials.gov NCT02314299 — include: The intervention record names this molecule or its validated development code. Eligibility here is registry identity matching; outcomes and applicability remain separately unappraised. Study families: NCT02314299.
  20. ClinicalTrials.gov NCT01518985 — include: The intervention record names this molecule or its validated development code. Eligibility here is registry identity matching; outcomes and applicability remain separately unappraised. Study families: NCT01518985.
  21. ClinicalTrials.gov NCT03323749 — include: The intervention record names this molecule or its validated development code. Eligibility here is registry identity matching; outcomes and applicability remain separately unappraised. Study families: NCT03323749.
  22. ClinicalTrials.gov NCT02653391 — include: The intervention record names this molecule or its validated development code. Eligibility here is registry identity matching; outcomes and applicability remain separately unappraised. Study families: NCT02653391.
  23. ClinicalTrials.gov NCT02367014 — include: The intervention record names this molecule or its validated development code. Eligibility here is registry identity matching; outcomes and applicability remain separately unappraised. Study families: NCT02367014.
  24. ClinicalTrials.gov NCT01786915 — include: The intervention record names this molecule or its validated development code. Eligibility here is registry identity matching; outcomes and applicability remain separately unappraised. Study families: NCT01786915.
  25. ClinicalTrials.gov NCT02245620 — include: The intervention record names this molecule or its validated development code. Eligibility here is registry identity matching; outcomes and applicability remain separately unappraised. Study families: NCT02245620.

FDA search

Exact active-ingredient terms in the openFDA Drugs@FDA endpoint, checked September 15. A zero result describes this indexed query only, not worldwide nonapproval or every FDA document.

1 retrieved · 1 included · 0 excluded · 0 awaiting a decision · 0 full-text appraisals

Search terms, record decisions and remaining work

products.active_ingredients.name:"ELAMIPRETIDE" OR products.active_ingredients.name:"ELAMIPRETIDE HYDROCHLORIDE"

Source retrievals and payload pins
  • Open the source endpoint ↗

    Retrieved 2026-09-15T08:54:11.534418+00:00 · HTTP 200 · SHA-256 80358dad70a79f9e7ac3b203f748bd6e1c119549f87a56aa61330ee39f27299d.

  • This query does not cover all FDA safety communications, labels, compounding proceedings or other regulatory systems.

Showing 1 of 1 record decisions, with included studies and unresolved records first. The versioned data inventory retains every record and study-family link.

  1. FDA NDA215244 — include: The application record contains the queried ingredient; indications, labels and approval documents require their own review.

EMA search

Local identity matching in the pinned EMA medicines metadata feed. This feed is not every national European authorization or every agency document; orphan designation is not marketing authorization.

0 retrieved · 0 included · 0 excluded · 0 awaiting a decision · 0 full-text appraisals

Search terms, record decisions and remaining work

Case-insensitive word-boundary matching of reviewed identity terms: ["elamipretide", "SS-31", "MTP-131", "Forzinity", "Bendavia"]

Source retrievals and payload pins
  • Open the source endpoint ↗

    Retrieved 2026-09-15T08:34:22.905972+00:00 · HTTP 200 · SHA-256 a947c2b8a56ac2b92ec96156843f262f5e516f83b375d6791d67a8f6dc695729.

  • Complete source-document appraisal and national European sources remain open.
  • Zero identity matches apply only to these feed fields and terms.

Showing 0 of 0 record decisions, with included studies and unresolved records first. The versioned data inventory retains every record and study-family link.

    EMA search

    Local identity matching in the pinned EMA documents metadata feed. This feed is not every national European authorization or every agency document; orphan designation is not marketing authorization.

    0 retrieved · 0 included · 0 excluded · 0 awaiting a decision · 0 full-text appraisals

    Search terms, record decisions and remaining work

    Case-insensitive word-boundary matching of reviewed identity terms: ["elamipretide", "SS-31", "MTP-131", "Forzinity", "Bendavia"]

    Source retrievals and payload pins
    • Open the source endpoint ↗

      Retrieved 2026-09-15T08:34:29.167460+00:00 · HTTP 200 · SHA-256 c0c68d7d77783e0355cbc75bdd04772e9acc163236fc68abcd794477c4c3d439.

    • Complete source-document appraisal and national European sources remain open.
    • Zero identity matches apply only to these feed fields and terms.

    Showing 0 of 0 record decisions, with included studies and unresolved records first. The versioned data inventory retains every record and study-family link.

      EMA search

      Local identity matching in the pinned EMA orphans metadata feed. This feed is not every national European authorization or every agency document; orphan designation is not marketing authorization.

      2 retrieved · 2 included · 0 excluded · 0 awaiting a decision · 0 full-text appraisals

      Search terms, record decisions and remaining work

      Case-insensitive word-boundary matching of reviewed identity terms: ["elamipretide", "SS-31", "MTP-131", "Forzinity", "Bendavia"]

      Source retrievals and payload pins
      • Open the source endpoint ↗

        Retrieved 2026-09-15T08:34:31.262344+00:00 · HTTP 200 · SHA-256 7448bf4e77e3345b3f3fbb7062c335d2302d8ea458a246bbe8256ce48aad2806.

      • Complete source-document appraisal and national European sources remain open.
      • Zero identity matches apply only to these feed fields and terms.
      • Direct orphan document pages were unavailable during review; only feed metadata is retained.

      Showing 2 of 2 record decisions, with included studies and unresolved records first. The versioned data inventory retains every record and study-family link.

      1. EMA EU/3/21/2430 — include: EMA orphan metadata identifies elamipretide for Treatment of Barth syndrome. This designation does not establish marketing authorization.
      2. EMA EU/3/22/2614 — include: EMA orphan metadata identifies elamipretide for Treatment of myopathic mitochondrial DNA depletion syndrome. This designation does not establish marketing authorization.
      Read the full Human Evidence Review

      Supervised synthesis

      What do selected human studies report about Elamipretide?

      Manually reviewed September 15, 2026 · version 1

      Browse all Human Evidence Reviews · Open preserved synthesis version 1 →

      Three selected reports cover the randomized Barth syndrome study, follow-up of the same cohort, and a separate primary mitochondrial myopathy trial. They do not cover every indication or replace the product-specific regulatory record.123

      Bottom line from this bounded source set

      The randomized Barth syndrome study did not meet its two primary endpoints; later uncontrolled follow-up reported improvements. The larger MMPOWER-3 trial in primary mitochondrial myopathy also missed its primary endpoints. These populations and study designs must be considered separately.123

      Reviewed study record

      TAZPOWER: randomized and early extension results

      Design

      A blinded placebo-controlled crossover trial was followed by an open-label extension.1

      Population

      Twelve participants with Barth syndrome entered the randomized study; ten entered the extension and eight reached 36 weeks.1

      Outcomes reported

      Neither primary endpoint was met in the randomized period. Walking distance and reported fatigue improved during the uncontrolled extension.1

      Limitations

      The extension lacks a concurrent placebo group and has attrition. Its changes cannot be interpreted as another randomized treatment effect; the abstract does not provide a detailed harms assessment.1

      Reviewed study record

      TAZPOWER: longer follow-up of the same cohort

      Design

      An open-label extension followed participants for up to 168 weeks.2

      Population

      Ten participants entered the extension; eight reached the week-168 visit.2

      Outcomes reported

      The authors reported improvements in walking distance and fatigue assessments. Injection-site reactions were the most common adverse events.2

      Limitations

      This is follow-up of TAZPOWER, not an independent trial. Selection, attrition and absence of concurrent controls limit attribution and generalizability.2

      Reviewed study record

      MMPOWER-3: primary mitochondrial myopathy

      Design

      A randomized, blinded, placebo-controlled phase 3 trial assessed 24-week outcomes.3

      Population

      The study randomized 218 participants with genetically confirmed primary mitochondrial myopathy.3

      Outcomes reported

      Neither walking distance nor fatigue met the primary efficacy endpoint. Most reported adverse events were mild or moderate.3

      Limitations

      This is a different disease population from Barth syndrome. Overall null results cannot be replaced by favorable exploratory subgroup findings.3

      Reviewed source set

      1. PubMed · 33077895A phase 2/3 randomized clinical trial followed by an open-label extension to evaluate the effectiveness of elamipretide in Barth syndrome, a genetic disorder of mitochondrial cardiolipin metabolism. ↗PubMed PMID 33077895 · EFetch XML reviewed 2026-09-15 · payload SHA-256 7be61a5476f72d7700d76f4e84d64de9269524a305e0f8591b3575519602e747
        No registered Crossref update in the reviewed snapshotChecked September 15, 2026 for DOI 10.1038/s41436-020-01006-8. This bounded result is not proof that the publication has never been corrected or retracted. Open the pinned Crossref work endpoint ↗
      2. PubMed · 38602181Long-term efficacy and safety of elamipretide in patients with Barth syndrome: 168-week open-label extension results of TAZPOWER. ↗PubMed PMID 38602181 · EFetch XML reviewed 2026-09-15 · payload SHA-256 675153339b38b362767882fe96e1930d9d3426f7eb3c18ee3219e019cffb9238
        No registered Crossref update in the reviewed snapshotChecked September 15, 2026 for DOI 10.1016/j.gim.2024.101138. This bounded result is not proof that the publication has never been corrected or retracted. Open the pinned Crossref work endpoint ↗
      3. PubMed · 37268435Efficacy and Safety of Elamipretide in Individuals With Primary Mitochondrial Myopathy: The MMPOWER-3 Randomized Clinical Trial. ↗PubMed PMID 37268435 · EFetch XML reviewed 2026-09-15 · payload SHA-256 ed6e6945e4e878261ab8d7868ae3118cae1354effbbf604ee9aeddfdce7df940
        No registered Crossref update in the reviewed snapshotChecked September 15, 2026 for DOI 10.1212/wnl.0000000000207402. This bounded result is not proof that the publication has never been corrected or retracted. Open the pinned Crossref work endpoint ↗
      Explore status sources, recent additions, and coverage gaps

      Living status brief

      What is the current status of Elamipretide?

      U.S. accelerated approval for a specified Barth syndrome population. FDA's September 19, 2025 approval letter requires a confirmatory trial to verify clinical benefit. The approval concerns this product and specified use; a recruiting trial record does not describe its full regulatory status.

      The cited status sources were checked through September 15, 2026. This answer changes only through the profile’s visible version and correction history.

      Evidence present

      What records are connected here?

      Evidence lanes
      3
      Source years
      8
      Drugs@FDA applications
      0

      These are counts in PeptideScanner’s selected public collection, not measures of research quality, medical relevance, safety, or effectiveness.

      Coverage gaps

      What remains unknown or unsupported?

      PeptideScanner currently has no selected safety records connected to this profile. That is a collection gap, not evidence that no such records exist.

      PeptideScanner does not infer an answer where the cited records do not provide one. Open the source-linked records below to inspect their scope and limits.

      Explore the Evidence Map and record counts

      Evidence Map v1

      What kinds of records connect to Elamipretide?

      Compare collection coverage →

      This map describes PeptideScanner’s selected, source-linked collection. It is not an evidence grade and does not establish research quality, safety, effectiveness, or medical relevance.

      50Selected dated records
      52Connected citations
      8Populated source years2013–2026
      3/4Record lanes present

      Registered human studies

      Trial composition

      4 of 9 selected trial records include at least one phase value; 4 of 9 include a status value in the retained structured contract.

      93 public history versions connect to 6 of these trial records across 11 submitted years. Open the record-history collection →

      1 exact source-declared trial-publication link connects selected NCT and PMID records. Open the exact graph →

      PhasesPhase 2 2Phase 3 1Phase 4 1
      Status at observationCompleted 2Active, not recruiting 1Recruiting 1

      A phase value is unavailable here for 5 selected records; this is not counted as “no phase.”

      A status value is unavailable here for 5 selected records; this is not counted as a trial status.

      PubMed bibliography

      Publication composition

      15 of 39 selected PubMed records include attributed title, venue, publication date, and publication-type fields.

      Publication typesJournal Article 14Research Support, Non-U.S. Gov't 8Review 4Research Support, N.I.H., Extramural 3Case Reports 2Randomized Controlled Trial 2Comparative Study 1Letter 1

      Bibliography fields unavailable here for 24 selected records; the records remain visible as metadata observations.

      Regulatory and publication surfaces

      What is structurally connected?

      Drugs@FDA applications
      0
      FDA Federal Register documents
      2
      DailyMed SPL versions
      0
      Canonical profile
      Published
      Profile structured data
      Published
      Linked dated records
      50

      Application counts reflect exact reviewed ingredient connections; Federal Register counts reflect exact reviewed peptide-name matches; DailyMed counts reflect listed SPL versions with retained ZIP receipts. “Published” describes this page’s indexing surface, not the state of scientific evidence.

      Profile publication history and record counts

      Exact identifier graph

      1 trial-publication link for Elamipretide

      Open the full graph →

      These links come only from structured NCT and PMID identifiers declared by PubMed or ClinicalTrials.gov. They do not summarize the publications or establish findings.

      Research hub

      Explore the Elamipretide evidence record

      Open all 50 records →
      50Dated records
      3Evidence types
      50Cited public sources
      8Source years

      Change ledger

      Recently changed for Elamipretide

      Open all 53 events →

      PeptideScanner change dates and cited source dates stay separate. These entries report collection or version activity, not a ranking of importance.

      Correction

      Elamipretide profile

      The previous profile described elamipretide only as investigational. We added FDA's September 19, 2025 accelerated approval of Forzinity for the specified Barth syndrome population. This correction does not extend that approval to other uses or formulations.

      PeptideScanner change date
      September 15, 2026
      Evidence lane
      Profile
      Record added

      ClinicalTrials.gov record NCT02693119

      PeptideScanner retained ClinicalTrials.gov record NCT02693119 at 2026-07-30T19:56:31.565Z. Its registry update date was 2021-11-30, and PeptideScanner's reviewed identity mapping was elamipretide.

      PeptideScanner change date
      September 9, 2026
      Cited source date
      November 30, 2021
      Evidence lane
      Clinical trials

      Official-source collections

      Source records for Elamipretide

      Open the full research atlas →
      PubMed39

      15 with attributed title, venue, publication-date, and type metadata.

      1. Targeting Mitochondria in MASLD: Comparative Evaluation of MitoQ and SS-31 (Elamipretide) in Aged Female Mice under Nutritional Stress.Physiological research · 2026 Aug 31
      2. Elamipretide: The first cardiolipin-directed mitochondrial therapeutic for Barth syndrome approved under accelerated approval.Drug discoveries & therapeutics · 2025 11 20
      3. Elamipretide in the Management of Barth Syndrome: Current Evidence and a Case Report.Molecular genetics and metabolism · 2025 08 11
      Explore all selected literature →
      ClinicalTrials.gov8

      93 public history versions across these dated registry records; status labels apply only to each cited observation.

      1. NCT07531251Registry update July 13, 2026 · current at observation
      2. NCT06373731Registry update May 20, 2026 · current at observation
      3. NCT05168774Registry update December 12, 2025 · completed at observation
      Explore all selected trial records →Explore all public record history →

      Counts and links describe PeptideScanner’s selected records. They are not rankings and do not establish research quality, safety, effectiveness, or medical relevance.

      Evidence types

      Coverage by source year

      Selected records span November 21, 2013 to September 8, 2026.

      Continue exploring

      Profiles with overlapping source-year coverage

      Open the research atlas →

      These links are based only on overlapping years and evidence lanes in PeptideScanner’s selected records. They do not imply that the peptides are medically or scientifically similar.

      Leuprolide8 shared source years215 dated records · 4 evidence typesLiraglutide8 shared source years167 dated records · 4 evidence typesOctreotide8 shared source years160 dated records · 4 evidence typesTeriparatide8 shared source years149 dated records · 3 evidence types

      These dates come from selected source records and PubMed metadata. They are not a complete or continuous timeline of research, safety, approval, or effectiveness.

      Evidence summary

      What the cited records say

      FDA's September 19, 2025 approval letter requires a confirmatory trial to verify clinical benefit. The approval concerns this product and specified use; a recruiting trial record does not describe its full regulatory status.

      This is a source summary, not a medical recommendation. Status may differ by jurisdiction and product.

      Cited context

      Related records

      Organizations named in cited records

      • Stealth BioTherapeutics Inc.

      Trial records

      Dated source history

      Updates connected to this profile

      Open the full cross-source timeline →
      1. Literature metadata

        Targeting Mitochondria in MASLD: Comparative Evaluation of MitoQ and SS-31 (Elamipretide) in Aged Female Mice under Nutritional Stress.

        PubMed lists PMID 42708867 in Physiological research as Journal Article, Comparative Study, with publication-date metadata of 2026-08-31 (day precision). PeptideScanner associated the retained record with the identity slug elamipretide. These fields reproduce attributed bibliographic metadata and do not summarize or endorse the article's findings.

      2. Literature metadata

        PubMed record PMID 42456009

        PeptideScanner retained PubMed bibliographic metadata for PMID 42456009 at 2026-08-10T18:13:55.284Z. PubMed recorded a metadata revision date of 2026-07-27 and an Entrez history date of 2026-07-15. PeptideScanner associated this retained record with the identity slug elamipretide. These dates describe PubMed metadata history, not the article's original publication date.

      3. Literature metadata

        PubMed record PMID 35611788

        PeptideScanner retained PubMed bibliographic metadata for PMID 35611788 at 2026-08-10T18:13:32.311Z. PubMed recorded a metadata revision date of 2026-07-26 and an Entrez history date of 2022-05-25. PeptideScanner associated this retained record with the identity slug elamipretide. These dates describe PubMed metadata history, not the article's original publication date.

      4. Literature metadata

        PubMed record PMID 42450582

        PeptideScanner retained PubMed bibliographic metadata for PMID 42450582 at 2026-08-10T18:13:55.269Z. PubMed recorded a metadata revision date of 2026-07-26 and an Entrez history date of 2026-07-15. PeptideScanner associated this retained record with the identity slug elamipretide. These dates describe PubMed metadata history, not the article's original publication date.

      5. Literature metadata

        PubMed record PMID 42219795

        PeptideScanner retained PubMed bibliographic metadata for PMID 42219795 at 2026-08-10T18:13:50.715Z. PubMed recorded a metadata revision date of 2026-07-17 and an Entrez history date of 2026-06-01. PeptideScanner associated this retained record with the identity slug elamipretide. These dates describe PubMed metadata history, not the article's original publication date.

      6. Literature metadata

        PubMed record PMID 42448476

        PeptideScanner retained PubMed bibliographic metadata for PMID 42448476 at 2026-08-10T18:13:55.255Z. PubMed recorded a metadata revision date of 2026-07-14 and an Entrez history date of 2026-07-14. PeptideScanner associated this retained record with the identity slug elamipretide. These dates describe PubMed metadata history, not the article's original publication date.

      7. Clinical trials

        ClinicalTrials.gov record NCT07531251

        PeptideScanner retained ClinicalTrials.gov record NCT07531251 at 2026-08-10T18:10:04.223Z. Its registry update date was 2026-07-13, and PeptideScanner's reviewed identity mapping was elamipretide.

      8. Literature metadata

        PubMed record PMID 42443448

        PeptideScanner retained PubMed bibliographic metadata for PMID 42443448 at 2026-08-10T18:13:55.249Z. PubMed recorded a metadata revision date of 2026-07-13 and an Entrez history date of 2026-07-13. PeptideScanner associated this retained record with the identity slug elamipretide. These dates describe PubMed metadata history, not the article's original publication date.

      9. Clinical trials

        Elamipretide Barth syndrome study record updated

        ClinicalTrials.gov lists a recruiting Elamipretide study sponsored by Stealth BioTherapeutics Inc.

      10. Literature metadata

        PubMed record PMID 41801306

        PeptideScanner retained PubMed bibliographic metadata for PMID 41801306 at 2026-08-10T18:13:44.801Z. PubMed recorded a metadata revision date of 2026-07-10 and an Entrez history date of 2026-03-09. PeptideScanner associated this retained record with the identity slug elamipretide. These dates describe PubMed metadata history, not the article's original publication date.

      11. Literature metadata

        PubMed record PMID 38237649

        PeptideScanner retained PubMed bibliographic metadata for PMID 38237649 at 2026-08-10T18:13:32.340Z. PubMed recorded a metadata revision date of 2026-07-09 and an Entrez history date of 2024-01-18. PeptideScanner associated this retained record with the identity slug elamipretide. These dates describe PubMed metadata history, not the article's original publication date.

      12. Literature metadata

        PubMed record PMID 42415111

        PeptideScanner retained PubMed bibliographic metadata for PMID 42415111 at 2026-08-10T18:13:53.859Z. PubMed recorded a metadata revision date of 2026-07-07 and an Entrez history date of 2026-07-07. PeptideScanner associated this retained record with the identity slug elamipretide. These dates describe PubMed metadata history, not the article's original publication date.