BridgeBio's Infigratinib is First Achondroplasia Therapy to Get FDA Breakthrough Status

BridgeBio Pharma, Inc., a biopharmaceutical company focusing on genetic diseases, recently announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation to their oral medication, infigratinib, for the treatment of children with achondroplasia. This Designation is intended to accelerate the development and review process for drugs that show significant improvement over existing therapies based on preliminary clinical evidence.

The Breakthrough Therapy Designation was awarded to infigratinib due to promising results from the PROPEL 2 clinical trial. Specifically, in Cohort 5 of the study, participants who received 0.25 mg/kg/day of infigratinib exhibited a statistically significant and sustained increase in their annualized height velocity (AHV). The results showed a mean change from baseline of +2.51 cm/year at Month 12, and +2.50 cm/year at Month 18, with both results having a p-value of 0.0015. Additionally, there was a significant improvement in body proportionality by Month 18, which carried a p-value of 0.001.

Adora Ndu, Chief Regulatory Affairs Officer at BridgeBio, expressed enthusiasm about the FDA's recognition, emphasizing the impact this could have on families affected by achondroplasia. She highlighted that the Breakthrough Therapy Designation supports the expedited development and regulatory review of infigratinib, potentially making it the first oral therapeutic option available for children with this condition.

Munira Shamim, founder of the U.S. advocacy group Growing Stronger, also commented on the significance of this milestone. She pointed out that achondroplasia affects more than just height, impacting overall quality of life and functionality. Shamim sees the FDA's decision as a crucial step toward providing families with more treatment options, thereby giving them greater control over their healthcare decisions.

BridgeBio is currently enrolling participants in PROPEL 3, a global Phase 3 study of infigratinib for achondroplasia. The company anticipates completing enrollment by the end of the year. Beyond achondroplasia, BridgeBio is also exploring the potential of infigratinib for treating hypochondroplasia and other skeletal dysplasias, conditions that present significant unmet medical needs.

Alongside the Breakthrough Therapy Designation, infigratinib has also received Orphan Drug Designation, Fast Track Designation, and Rare Pediatric Disease Designation from the FDA. If approved, BridgeBio may qualify for a Priority Review Voucher, which could further expedite the regulatory review process.

Founded in 2015, BridgeBio Pharma, Inc. is dedicated to discovering, developing, and delivering transformative medicines for patients with genetic diseases. Their pipeline includes a wide range of programs, from early-stage research to advanced clinical trials. The company's team is composed of seasoned drug developers and innovators who are committed to leveraging advances in genetic medicine to benefit patients swiftly.