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BridgeBio Pharma Inc. (BBIO) on Thursday reported encouraging phase-3 study data for its experimental oral therapy infigratinib, saying the drug improved growth outcomes in children with achondroplasia, a genetic form of dwarfism.
The topline findings stem from PROPEL 3, a global, yearlong Phase 3 trial that enrolled children ages 3 to younger than 18 with open growth plates. Participants were randomly assigned at a 2:1 ratio to receive infigratinib or placebo, with statistically significant improvements across key growth measures through Week 52.
The study met its main goal by demonstrating a meaningful increase in annualized height velocity compared with placebo. Children receiving the therapy grew faster on average, with a treatment benefit of more than 2 centimeters per year compared with placebo.
Secondary results supported the primary findings, including improvements in absolute growth rate and height scores relative to achondroplasia norms. The company said the magnitude of improvement ranks among the strongest seen in randomized studies for the condition.
Following the update, BridgeBio Pharma's stock traded over 17% higher in Thursday’s premarket. On Stocktwits, retail sentiment around the stock remained in ‘neutral’ territory amid ‘high’ message volume levels.
In an analysis of children younger than 8, infigratinib also achieved a statistically significant improvement in upper-to-lower body proportionality compared with placebo.
“In the broadest age range studied to date, oral infigratinib has demonstrated the highest and most significant improvement in annualized growth velocity, along with the first statistically significant improvement in body proportionality, in children aged 3 to 8 years, reported for any therapy approved or in development for this condition.”
-Ravi Savarirayan, Global Lead Investigator, PROPEL 3
BridgeBio said it intends to meet with regulators to discuss filing a New Drug Application in the U.S. and a Marketing Authorization Application in Europe in the second half of 2026.
BBIO stock has gained over 129% in the last 12 months.
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