BridgeBio Pharma's oral infigratinib has received Breakthrough Therapy Designation from the U.S. Food and Drug Administration (FDA) for the treatment of children with achondroplasia. This designation aims to expedite the development and review of drugs demonstrating substantial improvement over existing therapies for clinically significant endpoints.
The FDA's decision was based on preliminary clinical evidence from the Phase 2 PROPEL 2 trial. The trial evaluated infigratinib in children with achondroplasia, a genetic condition affecting bone and cartilage growth, leading to dwarfism and other health complications.
PROPEL 2 Trial Results
Data from Cohort 5 of the PROPEL 2 trial showed that infigratinib resulted in a statistically significant and sustained increase in annualized height velocity (AHV). Specifically, the mean change from baseline in AHV was +2.51 cm/year at 12 months and +2.50 cm/year at 18 months (p=0.0015). The trial also demonstrated statistically significant improvements in body proportionality at 18 months (p=0.001).
Potential Impact and Future Development
If approved, infigratinib could be the first oral treatment option available for children with achondroplasia. Current treatments are limited, and an oral therapy could significantly improve patient compliance and quality of life. BridgeBio is currently enrolling patients in the global Phase 3 PROPEL 3 study, with completion of enrollment anticipated by the end of the year. The company also plans to explore the potential of infigratinib in treating hypochondroplasia and other skeletal dysplasias.
Regulatory Advantages
In addition to Breakthrough Therapy Designation, infigratinib has received Orphan Drug Designation, Fast Track Designation, and Rare Pediatric Disease Designation from the FDA. These designations provide several benefits, including expedited development and review, potential for market exclusivity, and eligibility for a Priority Review Voucher upon approval.
Expert Commentary
"The receipt of Breakthrough Therapy Designation, the first ever for a treatment being developed for children with achondroplasia, marks another important milestone for our skeletal dysplasia program," said Adora Ndu, Pharm.D., J.D., Chief Regulatory Affairs Officer of BridgeBio. "This recognition by the FDA further confirms the strength of our Phase 2 data, and the substantial improvement on clinically significant endpoints over available therapies."
Munira Shamim, Founder of Growing Stronger, a U.S. advocacy group, added, "This action represents a significant step towards offering the first oral treatment option more readily for families living with achondroplasia. Having a wider range of therapies empowers families with greater control and choice over their healthcare decisions."
