BridgeBio Pharma Announces NEJM Publication of Phase 2 PROPEL 2 Study on Infigratinib for Achondroplasia in Children

BridgeBio Pharma, Inc. has reported promising 18-month results from the Phase 2 PROPEL 2 trial investigating the use of infigratinib for children with achondroplasia. These findings, which were detailed in the New England Journal of Medicine and presented at the European Society for Paediatric Endocrinology's annual meeting, highlight the potential efficacy and safety of this oral treatment.

Infigratinib is an experimental oral small molecule designed to inhibit FGFR3 signaling, targeting the root cause of achondroplasia. The recent data underscore its potential effectiveness and favorable safety profile as a pioneering oral treatment. A notable highlight from the study is the significant increase in annualized height velocity (AHV) in children receiving a daily dose of 0.25 mg/kg. Specifically, Cohort 5 showed a mean change in AHV of +2.50 cm/year at Month 18, with a highly significant p-value of 0.001. Additionally, the height Z-score improved by +0.54 from baseline, and body proportionality saw a statistically significant enhancement.

The PROPEL 2 trial, led by Dr. Ravi Savarirayan from the Murdoch Children’s Research Institute, has shown that infigratinib is well-tolerated over the 18-month period. No serious adverse events (SAE) or treatment-emergent adverse events (TEAEs) were reported, nor were there any negative changes in bone age or bone mineral density. This indicates the therapy’s potential for children with skeletal dysplasia.

The investigational drug has already received Breakthrough Therapy Designation from the U.S. Food and Drug Administration (FDA), recognizing its potential to substantially improve efficacy over existing treatments. Additionally, infigratinib has been granted Orphan Drug Designation, Fast Track Designation, and Rare Pediatric Disease Designation for achondroplasia by the FDA. If approved, BridgeBio may be eligible for a Priority Review Voucher.

The ongoing global Phase 3 PROPEL 3 trial continues to enroll patients, with completion expected by the end of 2024. This indicates the proactive steps being taken to further explore and validate infigratinib’s therapeutic potential.

Achondroplasia is the most prevalent form of disproportionate short stature, affecting around 55,000 individuals in the U.S. and the European Union. This genetic condition can lead to significant health and quality of life issues, including obstructive sleep apnea, middle ear dysfunction, kyphosis, and spinal stenosis. The condition is caused by mutations in the FGFR3 gene.

BridgeBio Pharma, a company dedicated to developing treatments for genetic diseases, was established in 2015. The company’s mission is to accelerate the development of innovative therapies for patients with genetic conditions through a pipeline ranging from early-stage research to advanced clinical trials.

The findings from the PROPEL 2 trial, coupled with the designations granted by the FDA, underscore the potential of infigratinib to become a transformative treatment for achondroplasia. BridgeBio remains committed to its mission of addressing unmet medical needs and improving the lives of those affected by genetic diseases.