Barth Syndrome Treatments
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Brand Name
Forzinity
Generic Name
Elamipretide
View Brand Information FDA approval date: October 30, 2025
Form: Injection
What is Forzinity (Elamipretide)?
FORZINITY is indicated to improve muscle strength in adult and pediatric patients with Barth syndrome weighing at least 30 kg. This indication is approved under accelerated approval based on an improvement in knee extensor muscle strength, an intermediate clinical endpoint. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial. FORZINITY™ is a mitochondrial cardiolipin binder indicated to improve muscle strength in adult and pediatric patients with Barth syndrome weighing at least 30 kg. This indication is approved under accelerated approval based on an improvement in knee extensor muscle strength, an intermediate clinical endpoint. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.
Approved To Treat
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Related Clinical Trials
Open-Label, Single-Arm Phase 2a Pilot Study to Evaluate the Safety and Tolerability of a Daily Subcutaneous Dose of Elamipretide in Older Adults
Summary: This study will utilize a daily dose of elamipetide for 4 weeks and will screen a sufficient number of subjects for 30 subjects to enroll in the study. The planned duration of the study includes baseline measurement, followed by 4 weeks intervention period, a midpoint (2week ±3days) check-in phone interview, and post intervention visits that mirror the baseline visits. After the last injection, su...