Talk Bio

Eli Lilly achieved another victory over Novo Nordisk with Zepbound outperforming CagriSema in a head-to-head trial sponsored by Novo 2 The FDA released draft guidance on its new Plausible Mechanism Pathway for personalized therapies targeting ultra-rare diseases, inspired by the success of Baby KJ's gene therapy case 1 2 The Plausible Mechanism Pathway focuses on treatments targeting root causes of rare conditions at cellular, genetic, or molecular levels 1 A bipartisan Senate hearing on rare disease drug authorization is scheduled for Thursday, with focus on FDA regulatory processes and treatment delays 2 3 The FDA's Rare Disease Week programming includes policy discussions on regulatory flexibility, patient access, and improved development pathways for rare disease therapies 3 Drugmakers and patient advocates expressed cautiously reserved judgment on the new guidance, citing recent FDA setbacks for cell and gene therapy candidates 1 FDA Commissioner Marty Makary...