BridgeBio's infigratinib gets FDA Priority Review for achondroplasia
The FDA set a target action date of February 4, 2027, and BridgeBio plans to file for approval in Europe before the end of 2026
Published
The FDA has accepted BridgeBio Pharma's New Drug Application for oral infigratinib in children with achondroplasia and granted it Priority Review. The agency's target action date, known as a PDUFA date, is February 4, 2027. BridgeBio says it is prepared to launch the drug in the US if it is approved.
Achondroplasia is the most common form of dwarfism, caused by an overactive FGFR3 protein that suppresses bone growth. Infigratinib is designed to block that protein directly. The two treatments now on the market, BioMarin's Voxzogo and Ascendis Pharma's Yuviwel, work differently, mimicking a hormone called CNP to counteract FGFR3 signalling, and both are given by injection. If approved, infigratinib would be the first oral therapy for the condition, according to Zacks Investment Research.
The application is backed by the Phase 3 PROPEL 3 trial, which met its main goal of improving annualized height velocity and a key secondary measure of height gain, according to GuruFocus. Zacks reported that participants treated with infigratinib for 52 weeks grew an average of 2.1 centimetres per year faster than those on placebo. BridgeBio first announced the FDA's acceptance and Priority Review designation on October 6, according to a company release carried by BioSpace.
Priority Review means the FDA aims to act within six months rather than the standard ten, cutting roughly four months from the usual timeline, Zacks reported. That faster path, paired with BridgeBio's statement that it is ready to launch, points to confidence in a positive outcome. It also comes amid a steady run of rare-disease approvals this quarter, including Mirum's Atebrioz and Scholar Rock's ISEMBYLD. BridgeBio's current lead product is Attruby, according to GuruFocus, and a favourable decision on infigratinib would give the company a second approved drug to sell. The planned European filing, due in the fourth quarter of 2026, would open a market for infigratinib before the US decision is even made.