BioMarin is discontinuing Phase 2 Voxzogo trials for Turner Syndrome, SHOX-deficiency and ACAN-deficiency after SCFE events in investigator-sponsored studies. Noonan syndrome and most ISS trials continue as planned.
FDA approves Ascendis Pharma's Yuviwel, the first once-weekly injection to boost growth in children 2+ with achondroplasia. Launch expected Q2 2026.
BridgeBio's oral infigratinib outperforms BioMarin's injectable therapy in boosting growth for kids with achondroplasia, marking a major win in treating the genetic cause of dwarfism.
Oppenheimer initiates coverage of Spruce Biosciences with an Outperform rating, citing strong FDA signals, accelerated approval potential, and possible acquisition interest from BioMarin.
BioMarin announces $4.8 billion acquisition of Amicus Therapeutics, adding rare disease treatments Galafold and Pombiliti to its portfolio with expected close in Q2 2026.
