PALO ALTO, Calif. — October 6, 2026
BridgeBio Pharma, Inc. announced that the U.S. Food and Drug Administration (FDA) has accepted its New Drug Application (NDA) for oral infigratinib for the treatment of children with achondroplasia and granted the application Priority Review. The FDA has assigned a PDUFA target action date of February 4, 2027, bringing BridgeBio’s investigational therapy closer to a potential U.S. regulatory decision. If approved, oral infigratinib could become the first approved oral therapy for children with achondroplasia, according to the company. BridgeBio said it is preparing for a potential U.S. launch following approval, making the NDA acceptance and Priority Review an important regulatory milestone for its skeletal dysplasia program. The company also plans to submit a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for achondroplasia in the fourth quarter of 2026.
PROPEL 3 Supports Oral Infigratinib Development
The FDA filing is supported by data 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 as well as its key secondary endpoint of change from baseline in height Z-score. BridgeBio reported an annualized height velocity treatment effect of up to 2.10 cm per year compared with placebo, with statistical significance reported at p<0.0001. In a pre-specified exploratory analysis involving children younger than eight years, oral infigratinib also demonstrated statistically significant improvements in body proportionality and arm span compared with placebo. The company reported a mean proportionality difference of -0.05 and an arm-span Z-score improvement of +0.37 standard deviations. BridgeBio also reported favorable trends in exploratory measures including sleep apnea and otitis media events following 52 weeks of treatment. The company said oral infigratinib was well tolerated in PROPEL 3, with no discontinuations or serious adverse events related to study treatment.
Oral Infigratinib Targets FGFR3 Signaling in Achondroplasia
Oral infigratinib is an investigational small-molecule therapy designed to inhibit FGFR3 signaling, a key biological driver of achondroplasia. Achondroplasia is caused by an activating variant in the FGFR3 gene, which leads to excessive signaling that affects growth plate development and results in disproportionate short stature. By reducing excessive FGFR3 activity, oral infigratinib is designed to address the underlying molecular pathway associated with skeletal growth abnormalities. BridgeBio said the therapy has received Breakthrough Therapy, Orphan Drug, Fast Track and Rare Pediatric Disease designations from the FDA, as well as Orphan Drug Designation from the EMA. The company is also evaluating oral infigratinib in other skeletal dysplasia programs, including hypochondroplasia, through ongoing clinical development efforts.
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BridgeBio Prepares for Potential U.S. Launch
The Priority Review brings BridgeBio’s oral infigratinib program into a key regulatory stage as the company prepares for a potential commercial launch in the United States. The company is developing the therapy as an oral targeted treatment intended to provide an additional option for children with achondroplasia, a genetic skeletal disorder that can also be associated with complications including obstructive sleep apnea, middle ear dysfunction, kyphosis and spinal stenosis. The FDA’s February 4, 2027 PDUFA date establishes the current target for regulatory action on the NDA. BridgeBio is continuing its broader skeletal dysplasia strategy through clinical programs in achondroplasia and hypochondroplasia, while also investigating potential effects of oral infigratinib beyond height and growth-related measures. The NDA acceptance and Priority Review mark a major regulatory milestone for BridgeBio as oral infigratinib advances toward a potential first approved oral treatment for children with achondroplasia.
Source::BridgeBio Pharma, press release
