Elamipretide

Article sent to my@email.address. All fields required; separate multiple recipients with a semicolon. Active subscribers access full article; others directed to abstract.

Stealth BioTherapeutics presented preclinical data on topical bevemipretide's protective effects in AMD models, showing improved mitochondrial function and reduced ROS production. The study involved rabbits, minipigs, and rats, demonstrating bevemipretide's tolerability and optimized delivery to the retina with low plasma concentrations. Topical treatment partially mitigated retinal thickness reduction in light-exposed rats and improved RPE cell viability. Stealth plans to initiate clinical studies for topical bevemipretide in dry AMD.

Recent review highlights advancements in Friedreich’s ataxia (FRDA) therapy, including the approval of Skyclarys (omaveloxolone) and emerging gene therapies, emphasizing the need for sensitive outcome measures and personalized medicine approaches.

Stealth BioTherapeutics announces FDA's advisory committee meeting on October 10, 2024, to evaluate elamipretide, a potential therapy for Barth syndrome, with a PDUFA action date of January 29, 2025. Elamipretide, a first-in-class mitochondria-targeted therapeutic, is also in Phase 3 trials for primary mitochondrial myopathy and dry age-related macular degeneration.

FDA to review Stealth BioTherapeutics' elamipretide for Barth syndrome on October 10, 2024, with a PDUFA date of January 29, 2025. Elamipretide, a mitochondria-targeted therapy, could be the first treatment for this ultra-rare disease affecting 150 individuals in the U.S.

Stealth BioTherapeutics announces FDA acceptance of New Drug Application for elamipretide, a potential treatment for Barth syndrome, supported by positive Phase 3 data. The FDA plans to hold an advisory committee meeting to discuss the application. Elamipretide has received Fast Track, Orphan Drug, and Rare Pediatric Disease designations.