Mississippi teen Christopher Pena has spent his life battling Barth syndrome, a rare genetic disorder. Now, a new drug is changing his life.

Mississippi teen Christopher Pena has spent his life battling Barth syndrome, a rare genetic disorder. Now, a new drug is changing his life.

Elamipretide gets FDA's blessing for ultra-rare pediatric mitochondrial cardioskeletal disease

Clinical and statistical reviewers favored a second complete response letter, but Hylton Joffe, director of the Office of Cardiology, Hematology, Endocrinology and Nephrology, said accelerated approva...

STRUCTURE: Elamipretide hydrochloride (formerly known as Bendavia, S-133, or MTP-131; marketed as FORZINITY) is an aromatic-cationic synthetic tetrapeptide and the first-in-class therapy for Barth syndrome (BTHS). The molecular formula is C32H49N9O5·3HCl, with a molecular weight of 749.2 g/mol. The chemical name is l-phenylalaninamide, d-arginyl-2,6-dimethyl-l-tyrosyl-l-lysyl-, hydrochloride (1:3). The peptide sequence, d-Arg-Dmt-Lys-Phe-NH2, contains the noncanonical amino acid Dmt [(S)-2-amino-3-(4-hydroxy-2,6-dimethylphenyl)propanoic acid].

Mitochondria serve an essential metabolic and energetic role in cellular activity, and their dysfunction has been implicated in a wide range of disorders, including cardiovascular conditions, neurodeg...

Mighty Therapeutics has dosed the first patient in 4TAZPower, its global Phase IV confirmatory study assessing Forzinity injection in those aged at least five years with Barth syndrome.

The U.S. Food and Drug Administration granted accelerated approval to Forzinity (elamipretide) injection as the first treatment for Barth syndrome, in patients weighing at least 30 kg.

Elamipretid ist in den USA zugelassen worden. Es ist die erste Therapie bei der ultraseltenen genetischen Erkrankung Barth-Syndrom.

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Le Forzinity, codéveloppé à l’IRCM par le Dr Peter Schiller, a été approuvé le 19 septembre dernier par la FDA contre le syndrome de Barth.

Streaming now at https://abc7.ws/484PqhJAn Orland Park baby's Forzinity treatment for a genetic disease, Barth Syndrome, was granted accelerated approval by ...

FDA approved Stealth BioTherapeutics' Barth syndrome drug Forzinity despite internal disagreement, with division director Joffe accepting higher uncertainty due to rare disease status.

The Pink Sheet’s Drug Review Profile looks at the clinical development and US FDA review timeline for Stealth's elamipretide for Barth syndrome.

NEEDHAM, Mass., Sept. 19, 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 that the U.S. Food and...

(MedPage Today) -- The FDA on Friday granted accelerated approval to elamipretide (Forzinity) as the first treatment for the ultra-rare Barth syndrome, which affects approximately 150 Americans. Elamipretide once-daily injections are now indicated... Source link