What's Happening?
BridgeBio Pharma's investigational drug, infigratinib, has demonstrated significant efficacy in a Phase 3 trial for treating achondroplasia, a common form of dwarfism. The PROPEL 3 study showed that infigratinib improved
yearly growth by 2.1 cm on average compared to placebo, meeting statistical significance. The drug also achieved key secondary endpoints, including body proportionality, without serious toxicities. These results pave the way for BridgeBio to file a new drug application with the FDA later this year.
Why It's Important?
The positive trial results for infigratinib represent a significant advancement in the treatment of achondroplasia, offering a potential new therapeutic option for patients. If approved, BridgeBio's drug could compete with BioMarin's Voxzogo, currently the only commercial therapy for this condition. The success of infigratinib could also enhance BridgeBio's market position and financial prospects, as well as provide a new standard of care for achondroplasia patients.
What's Next?
BridgeBio plans to meet with the FDA to discuss the regulatory pathway for infigratinib and expects to submit a new drug application in the second half of the year. The company is also conducting additional studies, including trials in newborns and toddlers with achondroplasia. The outcome of these regulatory and clinical efforts will determine the drug's future availability and impact on the treatment landscape for achondroplasia.
