Abstract
There was significant activity through December 2025 in respect of advancing new weight management therapies, with the first approved orally GLP-1 receptor agonist becoming available from Novo Nordisk, but with late stage and early phase assets being pursued by Lilly, Structure Therapeutics, Pfizer/YaoPharma, and Zealand/OTR. Cell and gene therapy for rare diseases was highlighted in new product approvals for Fondazione Telethon, Novartis, and positive clinical trial findings for Capricor Therapeutics, Kyverna Therapeutics, Encoded Therapeutics and Kite. Other rare disease therapies were the subject of announcements around deals and collaborations from BioMarin/Amicus, Mirum/Bluejay, Shionogi/Tanabe, Regeneron/Tessera, and positive clinical trial outcomes from Belie Bio. Novel delivery approaches progressed with the approval of Milestone's nasal spray formulation of a calcium channel blocker for PSVT and clinical trial progress for a doxorubicin microneedle array patch from Medicus for basal skin carcinoma.