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FDA Fast-Tracks Forzinity: First Barth Syndrome Treatment Approved

9/22/2025

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Summary

Story summary

The FDA has expedited approval for Forzinity (elamipretide), the first treatment for Barth syndrome, a rare mitochondrial disorder primarily affecting males.
This approval follows over a decade of research and addresses severe symptoms like heart failure and muscle weakness.
Forzinity enhances mitochondrial function and demonstrated positive results in muscle strength trials.
Patients must weigh at least 30 kilograms to qualify, and a post-approval trial is required to confirm long-term benefits.
Common side effects include mild to moderate injection site reactions.

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Story summary

The FDA has expedited approval for Forzinity (elamipretide), the first treatment for Barth syndrome, a rare mitochondrial disorder primarily affecting males.
This approval follows over a decade of research and addresses severe symptoms like heart failure and muscle weakness.
Forzinity enhances mitochondrial function and demonstrated positive results in muscle strength trials.
Patients must weigh at least 30 kilograms to qualify, and a post-approval trial is required to confirm long-term benefits.
Common side effects include mild to moderate injection site reactions.