Gene Editing
STAT+: BridgeBio’s treatment for genetic cause of dwarfism accelerates growth in small study
A novel drug for the most common cause of dwarfism accelerated children’s growth in a small but closely watched clinical trial, the company BridgeBio said Monday, advancing the latest treatment in what has been a polarizing field of study.
BridgeBio’s drug, a pill called infigratinib, increased the pace of growth by about 3 centimeters per year from baseline for a cohort of 10 children with achondroplasia, a rare genetic disorder that results in dwarfism and can lead to serious medical complications. That’s roughly double the observed benefit of Voxzogo, an injectable medicine from BioMarin Pharmaceutical that became the first approved achondroplasia treatment in 2021.
