The US FDA has accepted for Priority Review BridgeBio Pharma's (Nasdaq: BBIO) New Drug Application (NDA) for infigratinib, an oral FGFR1–3 tyrosine kinase inhibitor, for the treatment of children with achondroplasia, assigning a PDUFA target action date of February 4, 2027.
The NDA is supported by PROPEL 3, a global randomized, placebo-controlled Phase III study in children with achondroplasia that met its primary endpoint of change from baseline in annualized height velocity (AHV) at Week 52. The least-squares mean treatment difference versus placebo was +1.74 cm/year (95% CI 1.31–2.17; p<0.001), while the key secondary endpoint of change from baseline in height Z-score was also met.
In a prespecified exploratory analysis of children younger than 8 years, infigratinib demonstrated an improvement in body proportionality versus placebo, with a mean difference of −0.05 in upper-to-lower body segment ratio (p<0.05), while arm span Z-score improved by +0.37 SD (p<0.0001). No serious adverse events or treatment discontinuations were considered related to study drug. Results from PROPEL 3 were published in The New England Journal of Medicine.
Two pharmacologic treatments for achondroplasia are currently approved in the US: BioMarin's Voxzogo (vosoritide), a once-daily subcutaneous C-type natriuretic peptide analog, and Ascendis Pharma's YUVIWEL (navepegritide), a once-weekly subcutaneous CNP analog that received accelerated approval in February 2026 for children aged 2 years and older with open epiphyses. If approved, BridgeBio said infigratinib would be the first oral therapy for achondroplasia.