BridgeBio achondroplasia pill shows compelling growth and body proportionality benefit

BridgeBio Pharma has reported positive Phase 3 results for its oral infigratinib (BBP-831) in children with achondroplasia, demonstrating statistically significant increases in annualized height velocity and, importantly, the first statistically significant improvement in body proportionality against placebo in a randomized trial for this condition. The PROPEL 3 trial showed a mean treatment difference in annualized height velocity of +2.10 cm/year vs placebo at week 52, and a key secondary endpoint showed a statistically significant improvement in upper-to-lower body proportionality, with a least squares (LS) mean treatment difference of -0.05 (p<0.05) in children under 8 years. These results support the potential of infigratinib as a first oral, once-daily, FGFR3-targeted therapy for achondroplasia, with U.S. and EU regulatory filings planned in the second half of 2026 and a favorable safety profile to date. Earlier Phase 2 data also showed sustained height gains and improved body proportionality over 18 months, laying the foundation for the current Phase 3 findings.

Sources:

BridgeBio Reports Positive Phase 3 Topline Results for Oral ...

BridgeBio scores with dwarfism drug, setting up showdown with BioMarin, Ascendis

BridgeBio releases new phase 2 results for infigratinib for achondroplasia

Oral infigratinib boosts growth velocity in achondroplasia study

EX-99.1 - SEC.gov

FDA approves new treatment for acromegaly, a rare endocrine ...

BridgeBio Reports Durable Month 12 and 18 from Phase 2 ...

BridgeBio drug for genetic cause of dwarfism succeeds in key study

BridgeBio data show sustained benefit for achondroplasia ...

🆕 – #BridgeBio Announces First Child Dosed in ...

Oral Infigratinib Therapy in Children with Achondroplasia - PubMed

Achondroplasia - BridgeBio

NCT07169279 | Interventional Study of Infigratinib in Children < 3 ...

Achondroplasia Clinical Trial – PROPEL Program - BridgeBio