Infigratinib

Achondroplasia is a rare, genetic condition that affects bone growth. It is the most common type of skeletal dysplasia. Achondroplasia causes bone growth to slow down, especially in the long bones of the arms and legs. This causes short stature, shortened limbs, and other distinctive features. Genetic variants that cause fibroblast growth factor receptor 3 (FGFR3) overactivity can lead to skeletal conditions such as achondroplasia. QED Therapeutics are studying whether an investigational agent (infigratinib) has the potential to improve the irregular bone growth in achondroplasia by directly impacting FGFR3 overactivity, the underlying cause of the condition.

Infigratinib is not currently approved for the treatment of achondroplasia by the U.S. Food and Drug Administration (FDA) or any other health authority. It is the only oral agent under investigation for achondroplasia and is currently being studied in the PROPEL clinical program. The PROPEL clinical program is studying a wide range of outcomes related to growth, medical challenges, and quality of life in children and adolescents with achondroplasia. PROPEL is an observational study, in which no treatment is given. PROPEL 2 and PROPEL 3 are interventional studies, in which participants receive infigratinib and/or placebo. A placebo is a treatment with no active properties, such as a sugar pill. Participants from PROPEL 2 and PROPEL 3 have the option to join the PROPEL open-label extension study, where they may continue to receive treatment until their growth is complete.

Participants who complete at least 6 months in the PROPEL observational study may enroll in PROPEL 3, the Phase 3 investigational study of oral infigratinib sponsored by QED Therapeutics, a BridgeBio company.