Elamipretide, originally known as SS-31, is a mitochondrially targeted small molecule originally thought to be an antioxidant akin to plastiquinones (such as visomitin / SkQ1 which is approved for use in Russia), but which may primarily function through other mechanisms to improve mitochondrial function. As we should all know by now, mitochondrial function declines with age, and compensatory therapies that improve function may be at least modestly useful in a range of age-related conditions. So far, however, the available options (such as mitoQ and vitamin B3 derivatives like nicotinamide riboside) largely compare unfavorably to the benefits of exercise on mitochondrial function.
The recent FDA approval of elamipretide is for the treatment of a rare disease, as is often the case for new therapies with broad potential, as it is faster and easier to obtain approvals in rare disease indications. An approved therapy can be prescribed off-label for other uses, but it remains to be seen as to whether the community of anti-aging physicians develops a favorable view of elamipretide based on results in their patients, and whether the company is willing to manufacture enough of the drug for off-label use at this stage.
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 that the U.S. Food and Drug Administration (FDA) has granted accelerated approval to FORZINITY™ (elamipretide HCl) to improve muscle strength in adult and pediatric patients with Barth syndrome weighing at least 30 kilograms (kg) (approximately 66 pounds). Barth syndrome is a life-limiting pediatric mitochondrial cardioskeletal disease that affects approximately 150 individuals in the United States.
"The approval of FORZINITY, the first treatment option for Barth syndrome and the first FDA-approved mitochondria-targeted therapeutic, is a pivotal victory for the Barth syndrome community and offers hope for expedited regulatory attention to other ultra-rare diseases. We appreciate the FDA's close engagement in recent months and are grateful to the trial participants, caregivers, advocates, researchers and healthcare providers who persevered in partnership with us over this decade-long journey. We plan to continue providing expanded access to children weighing less than 30 kilograms who are currently receiving treatment or require emergency access, while we work with the FDA to generate data needed to expand the indication to include these children. We are committed to the continued development of therapies to treat all patients with Barth syndrome and other devastating diseases of mitochondrial dysfunction."
The approval of FORZINITY is supported by the efficacy and safety data from the TAZPOWER clinical trial. During the open-label portion of the TAZPOWER trial, knee extensor muscle strength improved from study baseline. The most common adverse reactions were injection site reactions which can be treated with oral antihistamines or topical corticosteroids. Continued approval for this indication may be contingent upon verification of clinical benefit in a confirmatory trial.
View the full article at FightAging
