Stealth provided a commercial and research update on FORZINITY (elamipretide) and its mitochondrial disease pipeline. FORZINITY is commercially available in the U.S. for eligible patients weighing at least 30 kg with Barth syndrome.
According to the company, 33 patients have initiated therapy, 85% of patients are fully covered, the average time to therapy initiation is less than 30 days, and 100% of eligible patients with FORZINITY prescriptions are enrolled in the Mito Assist patient support program. The company deployed a field medical team in early April and plans to launch its sales team in early May, with medical and commercial hiring nearly complete.
The company reports regulatory and clinical progress. Stealth said it has aligned with the U.S. Food and Drug Administration on a pathway to potential label expansion for patients under the approved weight threshold, including a one-month pharmacokinetic study planned this year to inform dosing and a potential supplemental NDA submission in 2027 for children as young as age 5.
For younger children and infants, the FDA encouraged continued access through an expanded access program and a registry to standardize data collection. The first site of a Phase 4 trial is open in Bristol, UK, and the company is meeting FDA post-marketing commitments.
Stealth noted its broader pipeline includes studies of elamipretide in dry age-related macular degeneration and POLG-related myopathy, development of bevemipretide for ophthalmic and neurological indications, and preclinical candidates SBT-589 and SBT-255. Reported adverse reactions for FORZINITY include injection site reactions and modest eosinophil elevations not linked to clinical symptoms.
