BridgeBio Pharma, Inc. reported positive Phase 3 topline results for oral infigratinib in children with achondroplasia, meeting primary and key secondary endpoints. The company intends to submit regulatory applications in the second half of 2026 and accelerate development for hypochondroplasia.
Key takeaways
PROPEL 3 Phase 3 trial for oral infigratinib in achondroplasia successfully met its primary endpoint of change from baseline in AHV at Week 52.
The trial successfully met its key secondary endpoint of change from baseline in height Z-score at Week 52.
Infigratinib demonstrated an LS mean decrease of -0.05 against placebo in upper-to-lower body proportionality at Week 52 (p<0.05).
BridgeBio intends to submit a New Drug Application (NDA) and Marketing Authorization Application (MAA) for infigratinib in the second half of 2026.
The company intends to accelerate development of infigratinib for hypochondroplasia and is enrolling participants in the observational run-in for the Phase 3 trial.
Infigratinib was well-tolerated with no discontinuations or serious adverse events related to study drug, and 3 cases (4%) of mild, transient hyperphosphatemia.