Sept. 06, 2023, positive feedback from the U.S. FDA and the EU EMA on key elements of the trial design for PROPEL3, its pivotal Phase 3 study of infigratinib in children with achondroplasia.
Achondroplasia is the most common cause of disproportionate short stature, affecting approximately 55,000 people in the United States and European Union, including up to 10,000 children and adolescents with open growth plates. Achondroplasia impacts overall health and quality of life, leading to medical complications such as obstructive sleep apnea, middle ear dysfunction, kyphosis, and spinal stenosis. The condition is uniformly caused by an activating mutation in FGFR3.
Infigratinib is an oral small molecule designed to inhibit FGFR3 and target achondroplasia at its source. It is incredibly exciting that infigratinib has the potential to become the first effective oral treatment option to improve growth, enhance functionality and decrease complications.