PALO ALTO, Calif. — September 9, 2026
BridgeBio Pharma, Inc. presented new exploratory analyses from the Phase 3 PROPEL 3 trial of oral infigratinib in children with achondroplasia, expanding the development story beyond its previously reported growth benefits. The data, presented at the 2026 Annual European Society for Paediatric Endocrinology (ESPE) Meeting, showed favorable trends in clinically meaningful complications including sleep apnea and otitis media, while longer-term findings from the PROPEL program continued to demonstrate improvements in growth and body proportionality. The findings strengthen BridgeBio’s strategy to position oral infigratinib as a potential treatment addressing broader medical consequences of achondroplasia rather than focusing solely on height. PROPEL 3 previously demonstrated a statistically significant +2.10 cm/year improvement in annualized height velocity versus placebo and significant improvement in body proportionality in children younger than eight years. The latest analyses add additional measures that may be important to families and clinicians when evaluating long-term treatment value.
Oral Infigratinib Shows Potential Beyond Growth Outcomes
The new PROPEL 3 analyses support a broader clinical development profile for oral infigratinib, with sleep-related and ear-related outcomes providing additional measures of potential benefit. At Week 52, the mean total Apnea-Hypopnea Index (AHI) increased by 10.4% from baseline in the oral infigratinib group compared with a 49.2% increase in placebo. Among children younger than eight years, mean total AHI remained unchanged with oral infigratinib, while increasing 63.2% with placebo. The estimated annualized rate of otitis media events was 38% lower with oral infigratinib compared with placebo, increasing to a 47% reduction among children younger than eight years. BridgeBio also reported favorable body-composition trends, including a smaller increase in BMI and visceral fat volume and a greater increase in lean body mass compared with placebo. These findings remain exploratory, but they expand the potential value proposition of FGFR3-targeted therapy by examining complications that can influence daily health, development and quality of life in children with achondroplasia.
Three-Year Data Strengthen Long-Term Development Strategy
Longer-term data from the PROPEL program further support sustained treatment effects through three years, providing important evidence as BridgeBio advances oral infigratinib toward potential commercialization. Children treated for up to three years demonstrated a +0.92 standard-deviation change from baseline in height Z-score at Year 3, while the upper-to-lower body segment ratio improved by 0.15 from baseline, indicating sustained gains in body proportionality. The company reported that oral infigratinib remained well tolerated over the longer treatment period, with no new safety signals identified. The broader PROPEL development program also includes PROPEL Infant & Toddler, an ongoing Phase 2/2b study in children under three years of age, supporting BridgeBio’s strategy of evaluating treatment earlier in the course of skeletal development. Additional development in hypochondroplasia further extends the potential application of the program, while qualitative research is being used to better understand the functional and medical impacts of related skeletal dysplasias.
Advertisement
BridgeBio Moves Toward Regulatory Review and Launch
BridgeBio has submitted a New Drug Application (NDA) to the U.S. FDA for oral infigratinib in achondroplasia and anticipates a U.S. launch in mid-2027, making regulatory execution and commercial preparation key next stages for the program. The company intends to submit a Marketing Authorization Application to the European Medicines Agency in the fourth quarter of 2026. Oral infigratinib has received FDA Breakthrough Therapy, Orphan Drug, Fast Track and Rare Pediatric Disease designations, supporting its regulatory positioning in a condition with significant unmet medical needs. If approved, BridgeBio believes the therapy could become the first approved oral targeted treatment directly addressing FGFR3 signaling in achondroplasia. The company is also advancing clinical development in younger children and hypochondroplasia, creating opportunities to broaden the program’s addressable population. With Phase 3 efficacy established and regulatory submissions underway, the latest PROPEL findings strengthen BridgeBio’s strategy to develop oral infigratinib as a potentially differentiated treatment designed to address both growth and broader medical complications associated with skeletal dysplasia.
Source: BridgeBio Pharma,press release
