Infigratinib achieved statistically significant improvement in growth rate and body composition in a Phase 3 clinical trial that tested the BridgeBio Pharma drug in achondroplasia, the most common form of dwarfism. Regulatory submissions are planned for later this year.
BioMarin Pharmaceutical’s Voxzogo is the first FDA approved therapy for achondroplasia, an inherited disorder that causes the most common form of dwarfism. With approvals in Europe and the U.S., and more regulatory decisions expected in 2022, the biotech said the drug has blockbuster potential.
Arbiter’s Anjali Jameson on hospital and payer alignment.
The Basel, Switzerland-based biotech company plans to trial the drug in achondroplasia patients next year.
Therachon has raised significant funding to support clinical proof of its soluable form of human fibroblast growth factor receptor 3. This could treat achondroplasia, a genetic mutation that is the leading cause of dwarfism.