BridgeBio has secured FDA Priority Review for its oral infigratinib NDA for children with achondroplasia, with a decision expected in February 2027.

FDA sets February 2027 decision date

The FDA has accepted BridgeBio Pharma’s New Drug Application (NDA) for oral infigratinib to treat children with achondroplasia and granted the application Priority Review.

The Prescription Drug User Fee Act (PDUFA) target action date is February 4, 2027. If approved, infigratinib would be the first approved oral treatment for children with achondroplasia in the US.

The application is supported by results from the Phase 3 Propel 3 study, which evaluated oral infigratinib in children with achondroplasia.

The trial met its primary endpoint, showing a statistically significant improvement in annualised height velocity at Week 52, as well as its key secondary endpoint assessing change from baseline in height Z-score.

BridgeBio said oral infigratinib was well tolerated in the study, with no treatment-related serious adverse events or discontinuations reported.

Infigratinib targets FGFR3 signalling

Achondroplasia is caused by activating variants in the FGFR3 gene, which lead to overactive signalling that disrupts growth plate development and results in disproportionate short stature.

Infigratinib is an oral small molecule designed to inhibit FGFR3 signalling. BridgeBio is also investigating the drug in hypochondroplasia and other skeletal dysplasia conditions.

In a pre-specified exploratory analysis of children younger than eight years, the company reported a statistically significant improvement in body proportionality compared with placebo.

BridgeBio has also reported exploratory findings involving sleep apnoea, ear infections and body composition, although these measures are not the basis of the current regulatory application.

Justin To, chief executive officer of BridgeBio Skeletal Dysplasias, said: “Being granted Priority Review means we are one step closer to potential FDA approval of the first oral treatment option for children with achondroplasia.”

“To that end, we are excited by the data we have generated on arm span, sleep apnea, ear infections, and proportionality, and we remain committed to further evaluate and share the impact of oral infigratinib on measures beyond growth,” To added.

European filing planned

BridgeBio said it plans to submit a Marketing Authorisation Application to the European Medicines Agency in the fourth quarter of 2026.

The company has received Breakthrough Therapy, Fast Track, Orphan Drug and Rare Pediatric Disease designations from the FDA for oral infigratinib in achondroplasia. The drug has also received Orphan Drug Designation from the EMA.

Achondroplasia is the most common cause of disproportionate short stature and can be associated with complications including obstructive sleep apnoea, middle ear dysfunction, kyphosis and spinal stenosis.

If approved, oral infigratinib would give families and clinicians another treatment option alongside existing approaches to managing achondroplasia.