Elamipretide Hydrochloride

iStock, Grandbrothers According to reporting from Reuters, reviewers at the agency pointed to an inability to differentiate from placebo to justify rejecting the drug, but an FDA office director approved the drug anyway. The FDA overruled its reviewers when approving Stealth Biotherapeutics’ Barth Syndrome drug, despite questions about the efficacy achieved in a 10-patient clinical trial, according to documents related to the approval. That approval came in September after Stealth had unsuccessfully tried to get elamipretide, now sold under the brand name Forzinity, across the finish line for years. Documents from the FDA show that Hylton Joffe, the FDA’s director of the office of cardiology, hematology, endocrinology and nephrology, signed off on the drug under the accelerated approval pathway. The approval was based on a 10-person study that showed the drug improved knee strength. However, reviewers at the FDA pointed out that the study, which was not placebo-controlled, did not directly link Forzinity to the patient improvements. Reviewers also pointed out that in the TAZPOWER trial, patients taking placebo improved about as much as those taking the drug in a test of how far they could walk in six minutes. Charu Gandotra, a cardiologist and FDA clinical team leader, also recommended against approving Forzinity to Joffe. The FDA’s actions were first reported Wednesday by Reuters . The feelings at the FDA seemed to be broadly mixed in parallel with the drug’s years-long journey to approval. The FDA declined to consider the drug for full approval back in 2021. In 2024, an advisory committee voted 10-6 in favor of supporting the drug for approval, though even the committee members who were in favor were equivocal in their support. “This was just impossible,” Gerard Berry, professor of Pediatrics at Harvard Medical School and one of the members who voted in favor of the drug, said at the time. “As a pediatrician and metabolic specialist who’s cared for these patients, to deprive somebody of being able to get the medicine that might help is just untenable for me.” Stealth was again rejected by the FDA in May, but the agency suggested the company resubmit under the accelerated pathway. Stealth did so, without submitting new data but addressing manufacturing and safety concerns brought up by the FDA in its prior review. Barth Syndrome is an ultra-rare disease affecting 150 people in the entire U.S. It arises from a genetic mutation affecting patients’ mitochondria, resulting in cardiac abnormalities that lead to muscle weakness and debilitating fatigue. Most patients die by the age of five. Forzinity is the first ever approved treatment for the condition. The Trump administration has repeatedly advocated for getting more approvals for rare diseases, and the Barth Syndrome patient community rallied to support Forzinity . “We have everything at stake in this,” Kate McCurdy, board chair at the Barth Syndrome Foundation, told BioSpace in September. “It’s not just hearsay or sort of wishful thinking that we see the benefits of this drug. We can see it.” This is not the first time that an FDA head has approved a contentious drug for a disease lacking treatment options. In 2023 , Peter Marks, the former head of the agency’s Center for Biologics Evaluation and Research, approved Sarepta Therapeutics’ Elevidys for Duchenne muscular dystrophy, despite recommendations from reviewers to decline the treatment.

Program underscores Stealth's commitment to patient access and partnership with the Barth syndrome community NEEDHAM, Mass., Oct. 9, 2025 /PRNewswire/ -- Stealth BioTherapeutics Inc. (the "Company" or "Stealth"), a commercial-stage biotechnology company focused on the discovery, development and commercialization of novel therapies for diseases involving mitochondrial dysfunction, today announced the launch of its Mito Assist™ Patient Support Program for access to FORZINITY™ (elamipretide) injection. FORZINITY was granted accelerated approval by the U.S. Food and Drug Administration (FDA) on September 19, 2025 as the first treatment for Barth syndrome, to improve muscle strength in adult and pediatric patients weighing at least 30 kilograms (kg). As part of this initiative, Stealth has selected AnovoRx Specialty Pharmacy ("AnovoRx") as its exclusive commercial distribution partner for FORZINITY in the United States. Through this partnership, AnovoRx will play a key role in helping patients, caregivers, and healthcare providers navigate the insurance coverage and access process. "We have been deeply committed to the Barth syndrome community for over a decade, and access to FORZINITY for eligible patients remains our highest priority," said Reenie McCarthy, Chief Executive Officer of Stealth BioTherapeutics. "We are investing significantly in our access infrastructure to make sure every eligible patient who may benefit from FORZINITY can receive it. Partnering with AnovoRx allows us to deliver a high-touch experience for families as they navigate access and coverage." Mito Assist™ will offer personalized patient support services to help affected individuals and caregivers understand coverage options, manage insurance submissions, and access financial assistance resources. This includes: Co-pay assistance for eligible commercially insured patients. Patient assistance programs for uninsured or underinsured individuals. Support for at-home subcutaneous injection training. Dedicated case management through AnovoRx to provide ongoing communication with patients, caregivers, and healthcare providers. Patients currently receiving elamipretide through Stealth's Expanded Access Program who meet the weight requirement of 30kg will be contacted directly by their healthcare providers and AnovoRx case managers to facilitate a seamless transition to commercial therapy once FORZINITY™ becomes available. Stealth anticipates that commercial product will be available through AnovoRx by December 2025, pending final distribution readiness and payer coverage activation. Patients and healthcare providers can learn more by calling 1-833-458-9099. About FORZINITY™ (elamipretide) injection INDICATION FORZINITY™ is a mitochondrial cardiolipin binder indicated to improve muscle strength in adult and pediatric patients with Barth syndrome weighing at least 30 kg. This indication is approved under accelerated approval based on an improvement in knee extensor muscle strength, an intermediate clinical endpoint. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s). CONTRAINDICATIONS Serious hypersensitivity to elamipretide or any of the ingredients. WARNINGS AND PRECAUTIONS FORZINITY is not approved in neonates. FORZINITY contains Benzyl Alcohol and serious adverse reactions including fatal reactions have been reported in low birth weight neonates and preterm neonates who received benzyl alcohol containing drugs intravenously. FORZINITY is not approved for intravenous use. Hypersensitivity reactions, including serious allergic reactions requiring emergency medical intervention, have been reported in patients receiving FORZINITY. Monitor patients for signs and symptoms of hypersensitivity reactions during treatment. ADVERSE REACTIONS Most common adverse reactions are injection site reactions. To report SUSPECTED ADVERSE REACTIONS, contact Stealth BioTherapeutics Inc. at 1-844-444-6486 or FDA at 1-800-FDA-1088 or . For more information about FORZINITY, please see the full US Prescribing Information. About Barth Syndrome Barth syndrome is an ultra-rare genetic condition characterized by mitochondrial abnormalities leading to exercise intolerance, muscle weakness, debilitating fatigue, heart failure, recurrent infections, and delayed growth. The disease is associated with reduced life expectancy, with 85% of early deaths occurring by age 5. Barth syndrome occurs primarily in males and is estimated to affect one in 1,000,000 males births or around 150 individuals in the United States. There are no EMA-approved therapies for patients with Barth syndrome. About Stealth BioTherapeutics Stealth BioTherapeutics' mission is to develop novel therapies to improve the lives of patients living with diseases of mitochondrial dysfunction. Stealth's commercial product, FORZINITY, was granted accelerated approval by the U.S. Food & Drug Administration (FDA) in September 2025 as the first FDA-approved treatment for Barth syndrome, as well as the first FDA-approved mitochondria-targeted therapeutic. Continued approval for this indication may be contingent upon verification of clinical benefit in confirmatory trial(s). Stealth is studying elamipretide in additional indications, including dry age-related macular degeneration and primary mitochondrial myopathy, and is developing its second-generation clinical-stage candidate, bevemipretide (SBT-272), for ophthalmic and neurological disease indications. Media Contact Ascent Strategic Communications Anna Stallmann [email protected] Investor Contact Precision AQ Austin Murtagh [email protected] Patient Advocacy [email protected] SOURCE Stealth BioTherapeutics Inc. WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED