Palo Alto, California – BridgeBio Pharma is positioning its oral achondroplasia therapy as a potential alternative to injectable treatments after late-stage data showed the drug not only increased growth but also improved body proportions in children—an outcome analysts view as a key competitive advantage.
The company said Sunday that it intends to submit infigratinib for regulatory approval in the third quarter, putting the drug on track for a potential commercial launch in early to mid-2027.
Results from the Phase 3 PROPEL 3 study demonstrated that children treated with once-daily infigratinib experienced a statistically significant improvement in body proportionality compared with placebo, according to a prespecified exploratory analysis. The trial enrolled more than 110 children with achondroplasia, who were randomly assigned to receive either the oral therapy or placebo.
The proportionality findings could distinguish infigratinib from currently available therapies, according to Jefferies. In a note to investors on Sunday, the firm said competing drugs have yet to show statistically significant improvements on this measure. BridgeBio likewise described the result as the first placebo-controlled demonstration of a statistically significant benefit in body proportionality for an achondroplasia treatment.
Beyond growth, analysts believe the endpoint could have important clinical implications. Jefferies said better body proportions may improve mobility and make everyday activities easier for patients, adding that it expects the outcome to be reflected in the drug’s eventual prescribing information.
The data were unveiled during a late-breaking presentation at the 2026 International Congress of Children’s Bone Health and simultaneously published in the New England Journal of Medicine.
Investigators also reported the study met its primary endpoint, with children receiving infigratinib growing 2.1 centimeters more per year than those given placebo. The result was consistent with topline data BridgeBio released in February.
Jefferies argued that an oral medicine offering both growth and proportionality benefits would represent a meaningful departure from the field’s injectable standards of care. The firm continues to project peak annual sales of roughly $1 billion for infigratinib in achondroplasia.
Achondroplasia is the most common form of dwarfism, affecting an estimated one in every 15,000 to 40,000 births. The inherited disorder disrupts bone growth and can lead to short stature, bowed legs, developmental delays, breathing complications and recurrent ear infections.
The treatment landscape currently consists of two FDA-approved injectable therapies. BioMarin’s daily therapy Voxzogo became the first approved treatment in 2021, while Ascendis entered the market earlier this year with the once-weekly injection Yuviwel.
The arrival of Yuviwel has intensified competition. Last October, BioMarin withdrew its target of reaching $4 billion in annual revenue by 2027, pointing in large part to mounting pressure on Voxzogo. The drug generated $220 million in first-quarter revenue, representing 3% year-over-year growth.
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