BridgeBio Pharma plans to file for approval in the third quarter. If granted, the oral drug could present a “highly differentiated” option over current achondroplasia therapies, according to Jefferies.
BridgeBio Pharma’s oral achondroplasia drug not only boosts growth in children but also significantly boosts body proportionality—a finding that analysts say could set the pill apart from competitors for treating the most common cause of dwarfism.
BridgeBio plans to file a regulatory package for infigratinib in the third quarter, with a potential launch “in early to mid 2027,” the company said Sunday.
In the Phase 3 PROPEL 3 study, BridgeBio enrolled more than 110 children with achondroplasia who were randomly assigned to receive a daily dose of the biotech’s drug or placebo. Results presented on Sunday showed that in a prespecified exploratory analysis, infigratinib led to significant improvements in the body proportion of patients compared to placebo.
This advantage in proportionality is “compelling,” Jefferies told investors in a note on Sunday, “since other players do not seem to hit stat-sig.” Indeed, BridgeBio claimed that infigratinib “achieved the first statistically significant improvement in body proportionality against placebo in achondroplasia.”
For Jefferies, the proportionality outcome could lead to broader benefits for patients. “We think proportionality drives meaningful outcomes in daily activities and mobility,” the analysts wrote. “We are optimistic proportionality will be included in the label.”
The data were presented as a late-breaker at the 2026 International Congress of Children’s Bone Health and were simultaneously published in the New England Journal of Medicine.
The presentation also included a readout for PROPEL 3’s primary efficacy endpoint, demonstrating a 2.1-cm/year advantage in annualized growth velocity in patients on infigratinib versus placebo—confirming topline data released in February.
“We think an oral option with proportionality benefits seems highly differentiated over the approved daily/weekly subQ” treatments for achondroplasia, Jefferies said. The firm conservatively estimates peak sales of $1 billion for infigratinib in achondroplasia.
Affecting one in 15,000 to 40,000 newborns, achondroplasia is a rare and genetic disorder that compromises bone development, in turn stunting growth in children. Other symptoms and complications include bowed legs, developmental delays, breathing problems and recurring infections.
Currently, there are two FDA-approved therapies available for patients. First to the market was BioMarin’s daily injection Voxzogo, which first won the regulator’s go-ahead in November 2021. Ascendis stepped up as a challenger in March, when the agency cleared the weekly therapy Yuviwel, also designed for subcutaneous delivery.
Ascendis appears to have caught BioMarin on the backfoot. In October 2025, BioMarin abandoned its previous objective of hitting $4 billion in revenue by 2027, a decision that stemmed largely from the competitive pressures on Voxzogo. The drug grew 3% year-on-year in the first quarter to bring in $220 million.
