BridgeBio is heading to regulators after its achondroplasia therapy, infigratinib, met its primary and secondary endpoints in a pivotal study.
During the Phase III PROPEL3 (NCT06164951) trial, a once-daily dose of infigratinib triggered a superior mean 2.10cm/year increase in annualised height velocity (AHV) from baseline over placebo – meeting the trial’s primary endpoint. The least squares (LS) mean treatment improvement was 1.74cm/year.
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Patients treated with the oral fibroblast growth factor receptor 1-3 blocker also experienced statistically significant improvements in absolute AHV at the 52-week mark compared with placebo. Within this period, the infigratinib cohort achieved growth of 5.96cm/year versus 4.22cm/year in the placebo group.
A pre-specified exploratory analysis in children under eight years of age, infigratinib also offered statistically significant improvements to upper-to-lower body proportionality from baseline at week 52 – prompting a LS mean decrease of 0.05 over placebo. This makes infigratinib the first drug to achieve this milestone in a randomised achondroplasia trial. However, this effect was not significant across the overall population, which achieved a mean LS treatment difference of -0.02 versus placebo.
Infigratinib was also found to be tolerable and safe, with no treatment-related discontinuations or serious adverse events (AEs) reported during the study. There were three mild cases of hyperphosphatemia – though these did not lead to dose reductions.
There were also no ophthalmic AEs related to FGFR1 or 2 inhibition or events synonymous with C-type natriuretic peptide (CNP) analogues like Voxzogo.
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By GlobalDataNext steps for infigratinib
Upon this positive readout, BridgeBio is looking to meet with regulators in H2 2026 to discuss next steps for new drug application (NDA) and marketing authorisation application (MAA) submission for infigratinib in achondroplasia – the most common form of dwarfism.
If infigratinib were to gain approval in the indication, it would join BioMarin’s daily injectable, Voxzogo (vosoritide) on the market – which became the first therapy to secure US approval in achondroplasia back in 2021. Voxzogo later got the greenlight for use in children under five. GlobalData, parent company of Clinical Trials Arena, estimates that Voxzogo will generate $1.3bn in sales during 2031.
Infigratinib may also come up against Ascendis Pharma’s once-weekly subcutaneous drug, TransCon CNP (navepegritidie), which remains in the pre-registrational stage after the US Food and Drug Administration (FDA) delayed its final decision on the drug in December 2025. Ascendis expects a verdict from the regulator on or before 28 February 2026. GlobalData currently estimates that TransCon CNP will post sales of $592m in 2031.
While infigratinib will be subject to competition, it may hold a dosing advantage over BioMarin and Ascendis’ rival therapies, as it can be administered as a once-daily pill rather than an injection.
According to BridgeBio’s CMO of skeletal dysplasia, Daniela Rogoff, the PROPEL3 data “support the potential of an oral medicine directly targeting FGFR3 overactivity… while fitting into daily life for families seeking a non-injectable option”.
If approved, GlobalData predicts that infigratinib will bring in sales of $598m in 2031.
