BridgeBio Pharma’s experimental drug for the most common cause of dwarfism accelerated children’s growth in a pivotal clinical trial, outperforming a rival medicine and clearing the way for the company to seek regulatory approval.
The drug, an oral medicine called infigratinib, met its primary goal in a study enrolling more than 100 kids with achondroplasia, a rare genetic disorder that results in short stature and can lead to serious medical complications. A daily dose of infigratinib sped up growth by an average of 2.1 centimeters per year compared to placebo, BridgeBio said in a statement. Adjusting for differences between the placebo and treatment groups, the difference was 1.74 centimeters per year. Both changes were statistically significant.
The top-line results surpass those seen with the only approved therapy for achondroplasia, an injectable from BioMarin Pharmaceutical, which accelerated growth by 1.57 centimeters per year in its pivotal study.
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