BridgeBio Pharma
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BridgeBio Pharma (BBIO) investor relations material

BridgeBio Pharma Study result summary

Complete event summary combining all related documents: earnings call transcript, report, and slide presentation.
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Study result summary12 Feb, 2026

Study background and design

  • PROPEL-3 was a global, randomized, double-blind, placebo-controlled phase III trial in 113 children aged 3 to <18 years with achondroplasia, evaluating oral infigratinib 0.25 mg/kg/day versus placebo over 52 weeks, the largest age range in any RCT for this condition.

  • Participants were randomized 2:1 to infigratinib or placebo after at least 6 months in an observational study, with an option for long-term extension.

  • The primary endpoint was change in annualized height velocity (AHV) at week 52; key secondary endpoints included change in height Z-score and body proportionality.

Efficacy results

  • Infigratinib achieved a mean difference in AHV of 2.1 cm/year and an LS mean difference of 1.74 cm/year vs. placebo at week 52 (p<0.0001), the largest seen in any RCT for achondroplasia.

  • The treatment arm reached a mean AHV of 5.96 cm/year, matching average-stature pediatric populations.

  • Statistically significant improvements were observed in height Z-score (LS mean increase of 0.41 SD, p<0.0001) and body proportionality (LS mean difference -0.05, p<0.05) in children under 8 years.

  • Efficacy was consistent across all age groups studied.

Safety and tolerability

  • Infigratinib was well-tolerated, with no serious adverse events, no discontinuations related to the drug, and no safety signals for FGFR1/2 inhibition.

  • Most adverse events were mild or moderate and balanced between arms; three cases of mild, transient hyperphosphatemia occurred, all resolving without intervention.

  • Mean serum phosphate levels remained within normal ranges throughout the study.

Significance of body proportionality improvement
Rationale for hypochondroplasia program acceleration
How does oral delivery differentiate infigratinib?
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Frequently asked questions

BridgeBio Pharma, Inc. engages in the discovery, development, and delivery of various medicines for genetic diseases. The company's lead candidates for the treatment of Fabry disease comprise a pipeline of recombinant enzyme-replacement therapies comprising elaprase and lysosomal-phospholipid-glycoprotein (PLG) analogs. It focuses on developing therapeutics for treating Fabry disease, a rare genetic disorder characterized by progressive loss of kidney function and the buildup of fatty substances that damage blood vessels and organs. BridgeBio Pharma, Inc.i s based in Waltham, Massachusetts.

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