BridgeBio’s oral small molecule infigratinib is proceeding with a late-stage study in children with achondroplasia after it improved body proportionality and height in children with dwarfism, the company announced Tuesday.
BridgeBio announced Tuesday that its drug candidate infigratinib showed positive Phase II results in treating children with achondroplasia, the most common form of dwarfism, and may have an edge over the current competition.
Infigratinib, an oral small molecule that inhibits FGFR3 signaling, targets the disease at its source and has been investigated in the Phase II PROPEL 2 trial. According to BridgeBio, the treatment sustained a statistically significant mean increase in annualized height velocity of +2.51 cm/year from the baseline at 12 months and +2.50 cm/year at 18 months, earning infigratinib a p-value of 0.0015.
BridgeBio also revealed that the drug candidate improved body proportionality from 2.02 at the baseline to 1.88 after 18 months, generating a p-value of 0.001. It was also shown to have a “well-tolerated” safety profile with no treatment-related adverse events being attributed to infigratinib.
“These data indicate that treatment with infigratinib is continuing to show increased growth velocity and improvements in body proportionality in children with achondroplasia,” Ravi Savarirayan, the global lead investigator for the PROPEL 2 trial, said in a statement. “This is encouraging and suggests that infigratinib has the potential to enhance functionality for people living with achondroplasia in addition to increasing growth.”
While BridgeBio will face competition in the achondroplasia market as the FDA approved BioMarin’s Voxzogo last year, some experts contend that infigratinib may have the advantage.
In a Tuesday note to investors, Bank of Montreal Capital Markets analysts said that infigratinib “will likely offer improved efficacy” versus Voxzogo. The analysts wrote that infigratinib had reached a better statistical significance for body proportionality and had a “clean safety” profile compared to BioMarin’s drug. They forecast that infigratinib could reach an estimated $1.4 billion in peak sales and may see an estimated 50% of Voxzogo patients possibly switching over.
For now, BridgeBio said it will continue with a Phase III trial in achondroplasia and finish enrollment by the end of the year. The company also aims to expand infigratinib’s development in hypochondroplasia, a form of short-limbed dwarfism, and has received “positive interactions” from the FDA and EMA to support a Phase II study in this indication.
“These results motivate us to continue evaluating infigratinib in other FGFR-related skeletal dysplasias and genetic conditions,” Daniela Rogoff, chief medical officer for skeletal dysplasias at BridgeBio, said in a statement. “The initiation of our observational study in hypochondroplasia and the obtainment of FDA and EMA alignment on the interventional study underlie our excitement for the potential of infigratinib as a treatment option for children with hypochondroplasia.”
Infigratinib has previously encountered regulatory hurdles. In May 2024, the accelerated approval for the treatment of metastatic cholangiocarcinoma was withdrawn at the sponsor’s request after difficulties were encountered in recruiting and enrolling for a confirmatory trial.
Tyler Patchen is a staff writer at BioSpace. You can reach him at tyler.patchen@biospace.com. Follow him on LinkedIn.
