BridgeBio Pharma announced on June 29 that its oral drug for achondroplasia not only increases growth in children but also significantly improves body proportionality, which analysts say could distinguish the treatment from competitors addressing the most common cause of dwarfism.
The company said it plans to submit a regulatory package for infigratinib in the third quarter, with a potential launch projected for early to mid-2027. In the Phase 3 PROPEL 3 study, more than 110 children with achondroplasia were randomly assigned to receive either a daily dose of infigratinib or placebo. Results presented on Sunday showed that a prespecified exploratory analysis found significant improvements in body proportion among patients who received the drug compared to those given placebo.
Jefferies told investors, “This advantage in proportionality is ‘compelling,’ since other players do not seem to hit stat-sig,” BridgeBio stated. Infigratinib “achieved the first statistically significant improvement in body proportionality against placebo in achondroplasia.” Jefferies added, “We think proportionality drives meaningful outcomes in daily activities and mobility. We are optimistic proportionality will be included in the label.”
The data were presented as a late-breaker at the 2026 International Congress of Children’s Bone Health and published simultaneously in the New England Journal of Medicine. The presentation also included results for PROPEL 3’s primary efficacy endpoint: patients on infigratinib had an annualized growth velocity advantage of 2.1 centimeters per year over those on placebo, confirming topline data released earlier this year.
Currently, two therapies approved by the Food and Drug Administration are available for patients: BioMarin’s daily injection Voxzogo and Ascendis Pharma’s weekly therapy Yuviwel, both delivered subcutaneously. BioMarin was first to market with Voxzogo following approval in November 2021; Ascendis followed with Yuviwel after agency clearance this March.
Achondroplasia affects one out of every 15,000 to 40,000 newborns and is caused by a rare genetic disorder that impairs bone development and stunts growth. Other symptoms include bowed legs, developmental delays, breathing problems and recurring infections.
