FORZINITY (elamipretide) — EAP, Phase IV sites & investigator studies
Mighty Therapeutics (formerly Stealth BioTherapeutics)
FORZINITY received FDA accelerated approval on Sept 19, 2025 — the first approved therapy for Barth syndrome, for adults and children ≥30 kg, and the first mitochondria-targeted therapeutic. Stealth BioTherapeutics became Mighty Therapeutics in around June 2026.
Three touchpoints for researchers and clinicians: the Expanded Access Program continues drug supply for patients who cannot access it through approval or trials, including <30 kg patients previously enrolled; a Phase IV confirmatory study is enrolling BTHS clinical sites, with the first patient dosed in 2026; and investigator-initiated studies have precedent, including an elamipretide IIS at CHOP, with drug supply negotiated case by case through medical affairs.
EAP: treating physicians of patients with serious mitochondrial disease and no comparable alternative. IIS: qualified academic investigators, case by case. Phase IV: BTHS clinical sites.
EAP: email EAP@mightytx.com and complete the Patient Information Intake Form (emergency line 617-600-6888). IIS and site-participation requests go to medical affairs via info@mightytx.com or the contact page; rolling.
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Last updated August 28, 2026 · Suggest an update to this entry