BridgeBio’s oral drug boosts growth in children with dwarfism in late-stage trial

BridgeBio Pharma said its experimental therapy boosted growth rates in children with a rare genetic disorder that causes dwarfism, sending the company’s shares 7% higher on Thursday.
The company said it plans to seek regulatory approval in the U.S. and Europe in the second half of 2026, and if approved, infigratinib would be the first oral therapy for achondroplasia.
In the late-stage trial, the therapy improved growth rate by 1.74 cm, on average, compared with placebo after 52 weeks in children with achondroplasia, a condition that causes disproportionate short stature.
In a pre-specified analysis of children aged 3 to 8 years, the therapy also showed statistically significant improvement in body proportions compared with placebo.
“We think these results clearly hit and exceed the bar hoped for by investors and make infigratinib highly competitive in the achondroplasia landscape as an oral with superior efficacy and clean safety,” said Barclays analyst Eliana Merle.
Currently, BioMarin Pharmaceutical’s injection, Voxzogo, is the only approved drug for the condition. Ascendis’ TransCon CNP is currently under review by the U.S. FDA.
A safe daily oral option is “always going to be the preferred way of administering medicine for young children,” which potentially is a “game changer” in this space, BridgeBio executive Justin To said.
The drug’s results outperformed Voxzogo and TransCon CNP, which showed mean annualized growth increases of 1.57 cm and 1.49 cm versus placebo, respectively, according to TD Cowen analyst Tyler Van Buren.
Achondroplasia affects about 55,000 people in the U.S. and the European Union, BridgeBio said.
The data positions infigratinib as a potential blockbuster, said Cantor analyst Josh Schimmer, who estimated the drug could generate nearly $2 billion in peak annual sales in achondroplasia.
The company is also testing the drug in another related condition called hypochondroplasia, a milder form of achondroplasia with less severe growth issues.