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Stealth BioTherapeutics Resubmits Drug Application for Barth Syndrome

8/21/2025

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Summary

Story summary

Stealth BioTherapeutics resubmitted its New Drug Application (NDA) for elamipretide, addressing FDA concerns from May 2025.
Barth syndrome, affecting approximately one in a million males worldwide, currently has no FDA-approved therapies.
Recent leadership changes at the FDA may complicate drug approval for rare diseases.
Families impacted by Barth syndrome emphasize the need for urgent treatment progress.

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Story summary

Stealth BioTherapeutics resubmitted its New Drug Application (NDA) for elamipretide, addressing FDA concerns from May 2025.
Barth syndrome, affecting approximately one in a million males worldwide, currently has no FDA-approved therapies.
Recent leadership changes at the FDA may complicate drug approval for rare diseases.
Families impacted by Barth syndrome emphasize the need for urgent treatment progress.