BridgeBio Pharma recently announced that the US Food and Drug Administration (FDA) has accepted for filing its New Drug Application (NDA) with Priority Review (PR) for oral infigratinib for the treatment of children with achondroplasia. The FDA has assigned a Prescription Drug User Fee Act (PDUFA) target action date of February 4, 2027, and BridgeBio is prepared to launch oral infigratinib upon approval.

Justin To, Chief Executive Officer, 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. We also understand that for many families, it’s about more than just height or having an oral option. 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. We are grateful to the families and investigators who have partnered with us, and we are moving with urgency alongside the FDA."

PROPEL 3, the global phase 3 pivotal study of oral infigratinib in children with achondroplasia, met its primary endpoint of change from baseline in Annualised Height Velocity (AHV) at week 52 (LS mean treatment difference of +1.74 cm/year; mean treatment difference of +2.10 cm/year; p<0.0001) and its key secondary endpoint of change from baseline in height Z-score (p<0.0001).

In a pre-specified exploratory analysis in children younger than 8 years (more than half of participants), oral infigratinib became the first therapeutic option to show a statistically significant improvement in body proportionality against placebo in a randomised achondroplasia trial.

Oral infigratinib was well tolerated, with no discontinuations or serious Adverse Events (AEs) related to study drug. These data were published as an original research article in The New England Journal of Medicine and simultaneously presented at the International Congress of Children’s Bone Health (ICCBH) 2026 in a late-breaking oral presentation.

Additionally, the company also shared emerging data for oral infigratinib at ESPE about improvements beyond height for people with achondroplasia, including stabilising sleep apnea measures, reducing rate of ear infections, and impacting body composition.

Michael Hughes, Chair—Biotech Industry Liaison Committee, Little People of America, said, “For children and families living with achondroplasia, today’s news represents meaningful progress toward potentially expanding the range of available options. Our community holds diverse priorities and perspectives, and what matters is that individuals and families have meaningful choices as they consider their own healthcare goals. This milestone brings us one step closer to potentially having another option for families to consider together with their health care providers. We appreciate BridgeBio’s continued engagement with the achondroplasia community and its efforts to incorporate community perspectives throughout the development process.”

BridgeBio intends to submit a Marketing Authorisation Application (MAA) for achondroplasia to the European Medicines Agency (EMA) in the fourth quarter of 2026.

Oral infigratinib has received Breakthrough Therapy Designation (BTD) from the FDA based on results from the PROPEL 2 clinical trial, which met the FDA’s requirement of potentially demonstrating substantial improvement in efficacy over available therapies on clinically significant endpoints. It is the only therapeutic option in development for achondroplasia to hold BTD. In addition, it has received Orphan Drug Designation (ODD), Fast Track Designation (FTD), and Rare Pediatric Disease Designation (RPDD) for achondroplasia from the FDA, as well as Orphan Drug Designation (ODD) from the EMA.