BridgeBio Pharma, a biopharmaceutical company focused on developing medicines for genetic conditions, has announced positive topline results from Propel 3, the global phase 3 pivotal study of oral infigratinib in children living with achondroplasia.
Achondroplasia is the most common cause of disproportionate short stature, affecting approximately 55,000 people in the US and European Union (EU), including up to 10,000 children and adolescents with open growth plates. Achondroplasia impacts overall health and quality of life, leading to medical complications such as obstructive sleep apnoea, middle ear dysfunction, kyphosis, and spinal stenosis. The condition is uniformly caused by an activating variant in FGFR3.
‘Infigratinib is the first oral therapy designed to target FGFR3 and directly address the underlying cause of achondroplasia. In the broadest age range studied to date, oral infigratinib has demonstrated the highest and most significant improvement in annualised growth velocity, along with the first statistically significant improvement in body proportionality, in children aged three to eight years, reported for any therapy approved or in development for this condition. Taken together, these best-in-class results highlight the transformative potential for infigratinib to address aspects of achondroplasia beyond linear height, and with a product administered orally.”
Propel 3 was a global, one-year, 2:1 randomised, double-blinded placebo-controlled clinical study, evaluating the efficacy and safety of infigratinib in children with achondroplasia aged three to <18 years with open growth plates.
Based on these results, BridgeBio intends to meet with regulatory authorities to discuss plans for submission of a New Drug Application (NDA) and Marketing Authorization Application (MAA) for infigratinib in the second half of 2026 to support approval.
The company also intends to accelerate the development of infigratinib for hypochondroplasia, and is enrolling participants in the observational run-in for the phase 3 trial.
