BridgeBio’s Infigratinib Granted FDA Breakthrough Designation for Achondroplasia Treatment

BridgeBio Pharma, Inc. (Nasdaq: BBIO) has announced that its oral treatment infigratinib, designed for children with achondroplasia, has been granted Breakthrough Therapy Designation by the U.S. Food and Drug Administration (FDA). Achondroplasia, the most common form of dwarfism, affects skeletal growth, leading to significant health and functional challenges. This designation is significant as it aims to expedite the development and review process of treatments that show substantial improvements over available therapies, providing new hope for affected families.

Infigratinib’s designation is based on promising data from the PROPEL 2 clinical trial. In Cohort 5 of the study, infigratinib demonstrated a significant increase in annualized height velocity (AHV), with participants seeing a mean growth increase of +2.51 cm/yr at 12 months and +2.50 cm/yr at 18 months (p=0.0015). Additionally, there were marked improvements in body proportionality at Month 18 (p=0.001). These results indicate infigratinib’s potential as a first-in-class oral therapeutic option for children with achondroplasia, a skeletal dysplasia that can significantly impact quality of life.

Adora Ndu, Pharm.D., J.D., Chief Regulatory Affairs Officer of BridgeBio, expressed excitement about the designation, stating, “This recognition from the FDA validates the strength of our Phase 2 data and underscores the importance of advancing this treatment for families dealing with achondroplasia. We look forward to collaborating closely with the FDA to bring this therapy to those who need it.”

Munira Shamim, founder of Growing Stronger, an advocacy group for individuals with achondroplasia, applauded the FDA’s decision, emphasizing the wider implications of having an oral treatment option available. “This is a significant step for families seeking more control over healthcare choices. Achondroplasia affects more than just height—it impacts health, functionality, and quality of life. Having new therapies means more options and hope for a better future,” she said.

BridgeBio’s Phase 3 trial of infigratinib, known as PROPEL 3, is already underway, with enrollment expected to conclude by the end of the year. The company is committed to further exploring infigratinib’s benefits not only for achondroplasia but also for other skeletal dysplasias such as hypochondroplasia, where unmet medical needs persist.

In addition to the Breakthrough Therapy Designation, infigratinib has also received Orphan Drug, Fast Track, and Rare Pediatric Disease designations from the FDA. If approved, BridgeBio may qualify for a Priority Review Voucher, which would expedite future drug review processes.

Founded in 2015, BridgeBio is a biopharmaceutical company dedicated to discovering and developing transformative treatments for genetic diseases. With its experienced team and wide-ranging pipeline, the company continues to make strides in genetic medicine.