Eli Lilly achieved another victory over Novo Nordisk with Zepbound outperforming CagriSema in a head-to-head trial sponsored by Novo2
The FDA released draft guidance on its new Plausible Mechanism Pathway for personalized therapies targeting ultra-rare diseases, inspired by the success of Baby KJ's gene therapy case12
The Plausible Mechanism Pathway focuses on treatments targeting root causes of rare conditions at cellular, genetic, or molecular levels1
A bipartisan Senate hearing on rare disease drug authorization is scheduled for Thursday, with focus on FDA regulatory processes and treatment delays23
The FDA's Rare Disease Week programming includes policy discussions on regulatory flexibility, patient access, and improved development pathways for rare disease therapies3
Drugmakers and patient advocates expressed cautiously reserved judgment on the new guidance, citing recent FDA setbacks for cell and gene therapy candidates1
FDA Commissioner Marty Makary's plausible mechanism guidance concept was first introduced in April 2025 but formalized during Rare Disease Week1
CDC leadership changes are contributing to ongoing organizational upheaval2
Sources:
1. https://www.politico.com/newsletters/prescription-pulse/2026/02/24/fda-rolls-out-new-rare-disease-drug-playbook-00793522
2. https://www.biospace.com/business/lilly-bests-novo-again-rare-disease-week-goes-regulatory-more-cdc-leadership-upheaval
3. https://www.thefdalawblog.com/2026/02/rare-disease-month-developments-part-3-the-ugly-just-kidding-see-you-at-rare-disease-week/