BridgeBio’s Infigratinib Demonstrates Sustained Efficacy in Phase 2 Trial for Achondroplasia Treatment
1. Sustained Efficacy: BridgeBio's infigratinib showed sustained and statistically significant increases in annualized height velocity (AHV) and improvements in body proportionality in children with achondroplasia in the Phase 2 Cohort 5 trial.
2. Phase 3 Enrollment: The company has already started a Phase 3 registrational study of infigratinib in achondroplasia, with completion expected by the end of 2024.
3. Hypochondroplasia Expansion: BridgeBio has also initiated an observational run-in study (ACCEL) for infigratinib in children with hypochondroplasia, a related skeletal dysplasia.
4. Competitive Landscape: Infigratinib is seen as a potential competitor to BioMarin's Voxzogo, another achondroplasia treatment, with analysts noting its potential for superior efficacy and safety.
5. Market Potential: There are an estimated 14,000 to 25,000 children in the U.S. and Europe who have either achondroplasia or hypochondroplasia and would be eligible for treatment.