BridgeBio Pharma, a biopharmaceutical company developing therapies for genetic conditions, has announced positive topline results from PROPEL 3, its global Phase 3 pivotal study of oral infigratinib in children with achondroplasia.
"Achondroplasia is a genetic condition driven by FGFR3 that affects more than stature alone, with consequences on physical functioning and independence that can impact widely over a person’s lifetime,” said Ravi Savarirayan, global lead investigator for PROPEL 3.
“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 annualized growth velocity, along with the first statistically significant improvement in body proportionality, in children aged 3 to 8 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 one-year, global, 2:1 randomized, double-blind, placebo-controlled trial evaluating children aged 3 to <18 with open growth plates. The drug-related discontinuations or serious adverse events were reported, and side effects were mild and transient.
“There remains a significant unmet need for therapeutic options that are effective, practical, and less invasive for children living with achondroplasia,” said Daniela Rogoff, Chief Medical Officer, Skeletal Dysplasia of BridgeBio.
“The PROPEL 3 data support the potential of an oral medicine directly targeting FGFR3 overactivity to address important clinical needs, while fitting into daily life for families who are seeking a non-injectable option.
"These results represent meaningful progress for those who have been waiting for a better approach, and we look forward to advancing this program towards global submissions. We would like to thank the study participants, their families, investigators, and study staff who trusted us and joined us on this journey.”
