New potential treatment in development for achondroplasia

QED Therapeutics is a biotechnology company focused on precision medicine for FGFR-driven disorders.

QED Tx is a subsidiary company of BridgeBio Pharma and is based in S. Francisco, USA. Currently, it is focused on the development of infigratinib (compound BGJ398), an orally-administered FGFR selective tyrosine kinase inhibitor, for patients with FGFR-driven cancers and pediatric skeletal dysplasias.

Infigratinib has demonstrated potential in pediatric skeletal dysplasias, including achondroplasia. In the early work published in the Journal of Clinical Investigation (Komlra-Ebri D et al. JCO 2016), researchers demonstrated that low doses of infigratinib corrected pathological hallmarks of achondroplasia in mouse models.

By reaching the source of this condition, infigratinib demonstrated greater preclinical improvement in mouse models than any previously tested intervention for achondroplasia.

Stage of development

QED Therapeutics is currently completing a preclinical program in achondroplasia, including further efficacy studies and a robust safety program. Pending results from this program, the company intend to begin clinical studies with infigratinib in patients with achondroplasia in 2019.

For more information visit QED Tx website


Note: this statement was kindly shared by the company for ALPE to present to the patients' community and organizations.