Stealth Biotherapeutics has shared updates on FORZINITY (elamipretide), a treatment currently available in the U.S. for individuals with Barth syndrome, a rare mitochondrial disease. So far, 33 patients have started using the therapy, with 85% receiving full insurance coverage. Patients typically begin treatment within 30 days of receiving their prescription, and all eligible patients are enrolled in the Mito Assist support program, which helps them navigate their treatment journey.

This development is significant for those affected by mitochondrial diseases, as FORZINITY may offer a new avenue for managing symptoms and improving quality of life. The company is also working on expanding access to younger patients, including children as young as five years old, with plans for a pharmacokinetic study aimed at determining appropriate dosing. This could potentially lead to a broader label expansion by 2027, making the treatment available to more individuals in need.

The research is still in the early stages, with ongoing clinical trials and regulatory discussions. A Phase 4 trial is underway in the UK, and the company is committed to fulfilling its obligations with the FDA. While the reports of side effects from FORZINITY include mild injection site reactions, they have not been linked to any serious clinical symptoms. Additionally, Stealth is exploring other uses for elamipretide in conditions like dry age-related macular degeneration and certain myopathies, which could further enhance treatment options for those with mitochondrial dysfunction.

Source: longevity.technology