Elamipretide official document review · version 1
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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.
- SPIBA-201 · NCT03098797 ↗ · FDA PDF pp. 85 ↗
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.