Summary

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• This summary is based on the review of two randomized controlled trials. ^[1-2]
• Forzinity (elamipretide) is indicated to improve muscle strength in adult and pediatric patients with Barth syndrome (BTHS) weighing at least 30 kg.
• In individuals with BTHS, elamipretide improved the 6-Minute Walk Test (6MWT) by 96.1 meters at week 168 in the open-label extension of the TAZPOWER trial (P = .003) and by +95.9 meters at 36 weeks in part 2 (P = 0.024).
• Barth Syndrome Symptom Assessment (BTHS-SA) scores improved, with mean Total Fatigue scores remaining below baseline at all open-label extension time points and a -2.1 point change at 36 weeks in part 2 (P = 0.031).
• Additional improvements were reported in knee extensor strength, cardiac volumes (left ventricular stroke, end-diastolic, and end-systolic), MLCL/CL biomarker values, and patient global impression of symptoms.
• In individuals with BTHS, elamipretide was well tolerated over the long term, with injection-site reactions reported as the most common adverse events. In a separate study of individuals with BTHS, safety outcomes and adverse events were not detailed in the study.
• All findings were derived exclusively from individuals with BTHS; elamipretide demonstrated significant improvements in 6MWT, BTHS-SA fatigue scores, muscle strength, cardiac parameters, and biomarkers in this population, with one study emphasizing long-term efficacy and safety and another highlighting efficacy and safety at 12 and 36 weeks.