BridgeBio’s oral infigratinib NDA for children with achondroplasia receives FDA Priority Review, with a February 4, 2027 PDUFA target date.
Written By: Creola Gonsalves, MS Biotech
Reviewed By: Pharmacally Editorial Team
BridgeBio Pharma 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 set February 4, 2027, as the Prescription Drug User Fee Act (PDUFA) target action date. BridgeBio said it is preparing for a potential launch if the application is approved.
If approved, oral infigratinib would become the first approved oral therapy for children with achondroplasia. The FDA action follows positive results from the Phase 3 PROPEL 3 trial, which evaluated once-daily oral infigratinib in children and adolescents aged 3 to under 18 years with achondroplasia.
PROPEL 3 Demonstrated Improvements in Growth
PROPEL 3 (NCT06164951) was a randomized, placebo-controlled Phase 3 study evaluating the efficacy and safety of oral infigratinib over 52 weeks. The trial met its primary endpoint, demonstrating a statistically significant improvement in annualized height velocity (AHV) from baseline at Week 52.
The least-squares mean treatment difference in AHV was +1.74 cm/year, while the mean difference was +2.10 cm/year, with p<0.0001. The study also met its key secondary endpoint, showing a statistically significant improvement in height Z-score.
The height Z-score improved by +0.41 standard deviations from baseline in the infigratinib group, with a treatment difference of +0.32 standard deviations versus placebo.
PROPEL 3 also demonstrated a statistically significant improvement in arm-span Z-score, with a treatment difference of +0.37 standard deviations versus placebo (p<0.0001). These findings support the potential of infigratinib to address aspects of skeletal growth beyond height velocity.
Body Proportionality and Potential Benefits Beyond Growth
A pre-specified exploratory analysis evaluated children younger than 8 years, who represented more than half of the study population. In this analysis, infigratinib demonstrated a statistically significant improvement in body proportionality compared with placebo.
The finding is notable because it represents the first reported statistically significant improvement in body proportionality in a Phase 3 achondroplasia trial.
BridgeBio also reported exploratory findings suggesting potential benefits beyond linear growth, including stabilization of sleep apnea measures and a reduction in the frequency of otitis media events.
At Week 52, the mean total Apnea-Hypopnea Index (AHI) increased by 10.4% from baseline in the infigratinib group compared with a 49.2% increase in the placebo group. Among children younger than 8 years, mean total AHI remained unchanged with infigratinib compared with a 63.2% increase with placebo.
The estimated annualized rate of otitis media events was 38% lower with infigratinib compared with placebo. In children younger than 8 years, the annualized rate was 47% lower with infigratinib. These findings were exploratory and should not be interpreted as established treatment effects for sleep apnea or recurrent ear infections.
Safety Findings
Oral infigratinib was generally well tolerated during the 52-week study period. No patients discontinued treatment because of a study-drug-related adverse event, and there were no serious adverse events considered related to the study drug.
Three patients, representing approximately 4% of the infigratinib group, experienced mild and transient hyperphosphatemia. These events did not require dose reduction or treatment discontinuation.
The safety findings will remain an important component of the FDA’s review as the agency evaluates the NDA.
Previous Regulatory Designations
Oral infigratinib previously received Breakthrough Therapy Designation from the FDA based on results from the PROPEL 2 clinical trial, which BridgeBio said demonstrated substantial improvement over available therapies on clinically significant endpoints.
The FDA has also granted infigratinib Orphan Drug Designation, Fast Track Designation and Rare Pediatric Disease Designation for achondroplasia.
BridgeBio intends to submit a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) in the fourth quarter of 2026.
Next Regulatory Milestone
The FDA’s acceptance of the NDA and Priority Review designation represent the next major regulatory milestone for BridgeBio’s oral infigratinib program. The February 4, 2027, PDUFA target date will determine the timing of the FDA’s decision on whether to approve the therapy for children with achondroplasia.
If approved, oral infigratinib could provide a new oral treatment option for children with achondroplasia, a population with a significant need for therapies that address abnormal skeletal growth and associated clinical complications.
Justin To, CEO of BridgeBio’s Skeletal Dysplasias business, said the Priority Review brings the company closer to a potential FDA approval of an oral treatment option for children with achondroplasia.
Reference
BridgeBio Announces FDA Acceptance and Priority Review of NDA for Oral Infigratinib for Children with Achondroplasia, BridgeBio, 06 October 2026
A Study to Evaluate the Efficacy and Safety of Infigratinib in Children and Adolescents With Achondroplasia (PROPEL3), ClinicalTrials.gov ID NCT06164951
About the Writer
Creola Gonsalves (Linkedin) is an M.S. Biotechnology postgraduate with a strong interest in clinical research, evidence interpretation, and healthcare writing, with a focus on translating life-science knowledge into meaningful real-world insights.
She is trained in Good Clinical Practice (GCP), clinical research principles, and critical interpretation of randomized clinical trials, with certifications from NIH and Stanford University.
Her research background in biotechnological applications and microbial research strengthens her ability to understand scientific evidence and develop clear, accurate, and research-driven healthcare content.