FDA Accepts BridgeBio's Infigratinib NDA For Priority Review
(RTTNews) - BridgeBio Pharma, Inc. (BBIO) said the FDA has accepted its New Drug Application (NDA) for oral infigratinib for the treatment of children with achondroplasia and granted Priority Review.
The FDA has assigned a PDUFA target action date of February 4, 2027. BridgeBio said it is preparing for a potential U.S. launch of oral infigratinib if approved.
The NDA is supported by results from the global Phase 3 PROPEL 3 study, which met its primary endpoint of change from baseline in annualized height velocity (AHV) at Week 52.
PROPEL 3 showed a treatment effect of +2.10 cm per year in AHV compared with placebo, with a statistically significant result of p0.0001. The study also met its key secondary endpoint of change from baseline in height Z-score.
In a prespecified analysis of children younger than 8 years, oral infigratinib also showed statistically significant improvements in body proportionality compared with placebo, as well as an improvement in arm span z-score.
Treatment with oral infigratinib for 52 weeks was well tolerated, with no discontinuations or serious adverse events considered related to the study drug.
BridgeBio also reported favorable trends in exploratory measures including sleep apnea and otitis media events, highlighting potential effects beyond growth in children with achondroplasia.
Oral infigratinib is an investigational small molecule designed to inhibit FGFR3 signaling, which is overactive in achondroplasia due to activating FGFR3 variants. The drug is intended to improve bone growth by reducing FGFR3 activity.
If approved, BridgeBio said oral infigratinib would be the first approved oral therapy for children with achondroplasia. The company plans to submit a Marketing Authorization Application to the European Medicines Agency in the fourth quarter of 2026.
Achondroplasia is caused by an activating FGFR3 variant and can be associated with complications including obstructive sleep apnea, middle ear dysfunction, kyphosis and spinal stenosis.
BBIO has traded between $52.75 and $93.42 over the past year.
BBIO shares Friday's closed at $66.14, up 1.65%. In the premarket, stock is up 3.42% at $68.40.
