– In Cohort 5 of PROPEL 2, daily oral treatment of infigratinib at 0.25mg/kg resulted in statistically significant and sustained increases in annualized height velocity (AHV), with a mean change from baseline of +2.50cm/year at Month 18 (P=0.001)
- Data also presented at European Society of Paediatric Endocrinology on November 18th at 10 am GMT
- To date, infigratinib has received Breakthrough Designation from the U.S. Food and Drug Administration for achondroplasia, as well as Orphan Drug Designation, Fast Track Designation, and Rare Pediatric Disease Designation
- PROPEL 3, the global Phase 3 registrational study of infigratinib in achondroplasia, continues to enroll on schedule, with enrollment completion anticipated by end of 2024
PALO ALTO, Calif., Nov. 18, 2024 (GLOBE NEWSWIRE) — BridgeBio Pharma, Inc. (Nasdaq: NASDAQ:) (BridgeBio or the Company), a new type of biopharmaceutical company focused on genetic diseases, announced that positive 18-month results from PROPEL 2, a Phase 2 trial of the investigational therapy infigratinib in children with achondroplasia, were published as an original research article in the New England Journal of Medicine (NEJM) today. Infigratinib is an investigational oral small molecule designed to inhibit FGFR3 signaling and target achondroplasia at its source. These data, which were also presented today at the 62nd Annual European Society for Paediatric Endocrinology (ESPE) Meeting in Liverpool, demonstrate continued potential best-in-class efficacy and an encouraging safety profile as a first-in-class oral treatment option.
Publication of results from PROPEL 2 in the New England Journal of Medicine and Breakthrough Therapy Designation of infigratinib by the FDA underscore the significance and importance of these data and the potential of this oral therapy to not only increase height, but more importantly, enhance functionality for children with achondroplasia, said Ravi Savarirayan, M.D., Ph.D., of Murdoch Children’s Research Institute in Melbourne, Australia, the global lead investigator for PROPEL 2 and lead author of the NEJM publication.
Key results from the PROPEL 2 dataset were presented today at ESPE in a talk titled Oral infigratinib for children with achondroplasia: Month 18 results from the PROPEL 2 study demonstrate safety and durability of treatment effect on linear growth with improved body proportionality by Melita Irving, M.D., a clinical geneticist at Guy’s and St Thomas’ NHS Foundation Trust, London, UK…
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