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Stealth BioTherapeutics Unveils Mito Assist Patient Support Program

Stealth BioTherapeutics Inc., a pioneering biotechnology firm focused on innovative therapies for mitochondrial diseases, has this week launched its Mito Assist™ Patient Support Program. This initiative aims to enhance patient access to its newly FDA-approved drug, FORZINITY™ (elamipretide), which has been recognized as the first treatment for Barth syndrome. As of September 19, 2025, FORZINITY™ has gained accelerated approval for improving muscle strength in patients aged over 30 kilograms, both adults and children.

In an effort to facilitate better distribution and accessibility of FORZINITY, Stealth BioTherapeutics has joined forces with AnovoRx Specialty Pharmacy. This exclusive collaboration is set to play a crucial role in simplifying the process for patients, caregivers, and healthcare providers as they navigate the complexities of insurance and access to this treatment.

Commitment to the Barth Syndrome Community

Reenie McCarthy, CEO of Stealth BioTherapeutics, emphasized the company’s long-standing dedication to the Barth syndrome community, stating, "For over a decade, we have been committed to ensuring access to FORZINITY for eligible patients, which remains our highest priority." The partnership with AnovoRx, according to McCarthy, will enable families to receive comprehensive support as they work through the insurance coverage process.

The Mito Assist™ program offers a range of personalized support services designed to empower affected individuals and their caregivers. Key components of the program include:

• - Co-pay assistance for those with commercial insurance, helping alleviate financial burdens.
• - Patient assistance programs for individuals who are uninsured or underinsured, ensuring that they do not miss out on essential treatments.
• - At-home subcutaneous injection training, which equips patients with the necessary skills and confidence to administer the treatment themselves.
• - Dedicated case management via AnovoRx, offering ongoing support and communication between patients, their caregivers, and healthcare providers.

As part of a seamless transition, patients currently receiving elamipretide through Stealth’s Expanded Access Program who meet the weight requirement will be contacted directly by their healthcare providers and AnovoRx case managers when FORZINITY™ becomes available.

Anticipating that the commercial product will be accessible through AnovoRx by December 2025, Stealth BioTherapeutics is currently focusing on finalizing distribution readiness and payer coverage activation. Patients and healthcare providers looking for more information can reach out to the dedicated hotline at 1-833-458-9099.

Overview of FORZINITY™ (elamipretide)

FORZINITY™ is specifically designed as a mitochondrial cardiolipin binder, approved to enhance muscle strength in Barth syndrome patients weighing over 30 kg. Its approval marks a significant milestone in the treatment landscape, granted under an accelerated process based on promising clinical endpoints, specifically an increase in knee extensor muscle strength. It is essential to note that continued approval may depend on further verification of clinical benefits through ongoing studies.

The therapy is contraindicated in cases of serious hypersensitivity to elamipretide or its components, and is not approved for use in neonates due to severe risks associated with Benzyl Alcohol.

Understanding Barth Syndrome

Barth syndrome is an ultra-rare genetic disorder mostly affecting males, characterized by mitochondrial dysfunction that leads to severe health challenges including muscle weakness, growth delays, heart failure, and increased susceptibility to infections. The condition notably reduces life expectancy, with a staggering 85% of early fatalities occurring before the age of five. Although it is estimated to impact one in every million male births, the lack of approved treatments underscores the importance of the recent developments surrounding FORZINITY.

Stealth BioTherapeutics continues to champion advancements in mitochondrial therapy, with ongoing research geared towards additional therapeutic areas including age-related macular degeneration and primary mitochondrial myopathy. Moreover, they are developing their follow-up candidate, bevemipretide (SBT-272), for various neurological and ophthalmic conditions.

For more detailed information on FORZINITY or the Mito Assist Program, stakeholders are encouraged to refer to the official resources provided by Stealth BioTherapeutics.