BioMarin Plans FDA Submission After Positive VOXZOGO Hypochondroplasia Trial
BioMarin’s VOXZOGO improved growth in children with hypochondroplasia, showing strong trial results and good safety.
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BioMarin Halts Voxzogo Phase 2 Trials in Turner Syndrome, SHOX Deficiency & ACAN Deficiency After SCFE Safety Events
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.
Ascendis Pharma Scores FDA Approval for Yuviwel – Weekly Injection to Boost Growth in Achondroplasia
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 Breakthrough: Oral Drug Succeeds in Dwarfism Study
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 Starts Coverage on Spruce Biosciences with Bullish Outlook
Oppenheimer initiates coverage of Spruce Biosciences with an Outperform rating, citing strong FDA signals, accelerated approval potential, and possible acquisition interest from BioMarin.
BioMarin to Acquire Amicus Therapeutics in $4.8 Billion Deal
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.