This is a Phase 2, multicenter, open-label, dose-escalation and dose-expansion study to evaluate the safety, tolerability, and efficacy of infigratinib, a fibroblast growth factor receptor (FGFR) 1-3-selective tyrosine kinase inhibitor, in children 3 to 11 years of age with Achondroplasia (ACH) who previously participated in the PROPEL study (Protocol QBGJ398-001) for at least 6 months. The study includes dose escalation with extended treatment, and dose expansion. Click here to review the study.
QED Therapeutics, Inc. (“QED”), a subsidiary of BridgeBio Pharma, Inc. (“BridgeBio”) focuses on bringing medicine to people living with conditions that are caused by changes in fibroblast growth factor receptor (FGFR) genes. QED is developing a medicine which may improve the overall health and quality of life of people with achondroplasia. This investigational medicine is called infigratinib (formerly called BGJ398) and may be an option for children to take while they are still growing. Infigratinib is a small tablet taken by mouth that works by reducing the activity of proteins called fibroblast growth factor receptors, or FGFRs. Because increased activity in one of the FGFRs – FGFR3 – leads to…