BridgeBio’s Achondroplasia Data Portend Competition With BioMarin’s Voxzogo

Less than a year after a reorganization that included a headcount reduction and consolidation of sites, BridgeBio Pharma, Inc. may be on the upswing again as it reported Phase II data on 6 March for its achondroplasia candidate infigratinib that suggest superior efficacy to BioMarin Pharmaceutical Inc.’s Voxzogo (vosoritide) in improving growth rates for children with the genetic disorder also known as dwarfism. BridgeBio’s share price soared after the pre-market data release.

In the fifth cohort of the Phase II PROPEL2 study of the oral FGFR3 inhibitor, BridgeBio and portfolio company QED Therapeutics noted that infigratinib produced an 80% response rate – defined as at least a 25% change from baseline in annualized height velocity (AHV) – in 10 of 12 evaluable patients in the cohort. This portion of the study is testing the highest dosage being used, 0.25mg/kg daily, and showed a mean increase of 3.03cm at six months of therapy (p=0.0022)