Elamipretide

Katherine Talcott, MD, discusses potential therapies for geographic atrophy (GA) secondary to age-related macular degeneration (AMD) at OSN New York 2024, highlighting ANX007, Danicopan ALXN 2040, AVD-104, JNJ-1887, ASP7317, RG6501, elamipretide, Glideuretinal, AREDS/AREDS2, and ONL1204. ANX007 showed significant protection from vision loss in the ARCHER Trial, while Danicopan ALXN 2040 is an oral agent inhibiting complement factor D. AVD-104 demonstrated visual acuity gains in the SIGLEC study. Gene therapies like JNJ-1887 and ASP7317 aim to protect retinal cells. Elamipretide showed positive effects on visual function in the ReCLAIM study. Glideuretinal and AREDS/AREDS2 are antioxidants, and ONL1204 is a Fas inhibitor reducing retinal cell apoptosis.

FDA considers removing oral phenylephrine from OTC guidelines due to lack of efficacy, not safety concerns. Public comments invited until May 7, 2025, before final decision. Phenylephrine is in many nasal decongestants like Sudafed and Mucinex. FDA's proposal does not affect nasal spray decongestants.

FDA advisory committee voted 10-6 in favor of Stealth Biotherapeutics’ elamipretide for Barth syndrome, despite the SPIBA-201 trial failing to meet its primary endpoint. Patients and doctors advocated for the drug's approval, citing improvements in weight, strength, and stamina. The FDA is expected to decide on the new drug application in January 2025.

Stealth BioTherapeutics announces FDA advisory committee's 10-6 vote in favor of elamipretide for treating Barth syndrome, based on studies like TAZPOWER and SPIBA-001. If approved, elamipretide would be the first therapy for this rare, life-shortening disease affecting ~150 US individuals. The FDA decision is expected by January 29, 2025.

FDA advisors voted 10-6 for elamipretide as a treatment for Barth syndrome, despite limited evidence. The decision was influenced by the drug's potential benefits and the rarity of the disease, which makes large-scale trials difficult.

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The FDA’s Cardiovascular and Renal Drugs Advisory Committee voted 10–6 to support Stealth BioTherapeutics’ elamipretide for Barth syndrome, despite concerns over data quality and clinical efficacy. Panelists acknowledged the urgent need for treatment in rare diseases, with some citing patient testimonials as compelling. The FDA remains skeptical, questioning the drug’s approvability and its reliance on surrogate biomarkers.

FDA advisory committee voted 10 to 6 in favor of elamipretide for treating Barth syndrome, with a PDUFA action date of January 29, 2025. If approved, elamipretide would be the first therapy for this ultra-rare, life-threatening mitochondrial disease affecting about 150 individuals in the U.S.

The FDA raised doubts about Stealth BioTherapeutics' elamipretide data for Barth syndrome treatment, citing failure in primary endpoints of TAZPOWER trial. The agency also questioned the adequacy of open-label extension and externally-controlled trials, and deemed the proposed postmarketing plan insufficient. The Cardiovascular and Renal Drugs Advisory Committee will discuss the data package and accelerated approval on Thursday.