How many drugs treat Barth syndrome?

Drug Landscape tracks 1 marketed treatments for Barth syndrome (deduplicated by molecule). 0 Phase 3 candidates and 0 Phase 2 candidates are in the pipeline.

How do I subscribe to ongoing alerts?

Subscribe to /feed/disease/barth-syndrome.xml in Feedly / Inoreader. Every regulatory action, PDUFA date, or trial completion in this treatment area lands in your feed automatically.

Last reviewed 2026-06-10 · How we verify

Barth syndrome — Treatment Landscape & Competitive Intelligence

Name: Drug Landscape Pharmaceutical Database
Creator: Drug Landscape
Published: 2026-01-01
License: https://creativecommons.org/licenses/by/4.0/

Barth syndrome (disease) competitive landscape: 1 marketed treatments tracked, 0 Phase 3 candidates, 0 Phase 2 candidates. Recent regulatory actions and upcoming PDUFA dates across the entire treatment set.

1 marketed 0 Phase 3 0 Phase 2 Live · 30-min refresh

Marketed treatment landscape

Approved drugs treating Barth syndrome, deduplicated by molecule. See the full disease page for line-of-therapy detail.

Drug	Generic	Sponsor	Class	Target	Line of therapy	First approval
Forzinity	ELAMIPRETIDE	Stealth Biotheraps				2025-01-01

Phase 3 pipeline

No Phase 3 pipeline candidates tracked.

Phase 2 pipeline

No Phase 2 pipeline candidates tracked.

Recent regulatory actions (last 90 days)

No regulatory actions in the last 90 days for this treatment set.

Upcoming PDUFA dates (next 180 days)

No PDUFA dates in the next 180 days for this treatment set.

Sponsor landscape

Stealth Biotheraps · 1 drug in Barth syndrome

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Every new regulatory action, PDUFA date, or trial completion in Barth syndrome:

Cite this brief

Drug Landscape (2026). Barth syndrome — Treatment Landscape & Competitive Intelligence Brief. https://druglandscape.com/ci/disease/barth-syndrome. Accessed 2026-06-10.

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Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing