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FDA Rejects Stealth BioTherapeutics' Drug for Barth Syndrome

8/19/2025

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Summary

Story summary

Stealth BioTherapeutics' drug application for Barth syndrome was rejected by the FDA due to inadequate efficacy data.
The company seeks to highlight the difficulties in treating ultra-rare diseases.
Disagreements persist between Stealth and the FDA regarding clinical trial data requirements.
Stealth cautions that it may shut down if the FDA delays additional review.

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Story summary

Stealth BioTherapeutics' drug application for Barth syndrome was rejected by the FDA due to inadequate efficacy data.
The company seeks to highlight the difficulties in treating ultra-rare diseases.
Disagreements persist between Stealth and the FDA regarding clinical trial data requirements.
Stealth cautions that it may shut down if the FDA delays additional review.