Daniela Rogoff, MD, PhD, Vice President of Clinical Development at QED Therapeutics, a BridgeBio Company, discusses the phase 2 clinical trial testing infigratinib to treat achondroplasia.
Achondroplasia is a skeletal dysplasia characterized by the failure of normal conversion of cartilage into bone, most notably in longer bones, which results in disproportionate short stature. Achondroplasia is caused by a mutation in the FGFR3 gene, which causes the FGFR3 protein to be overly active. People with achondroplasia can experience serious health complications, including foramen magnum compression, spinal stenosis, sleep apnea, bowed legs, mid-face hypoplasia, permanent sway of the lower back, recurrent ear infections, and long term chronic pain.
One drug in development to treat achondroplasia is infigratinib, a kinase inhibitor that targets FGFR3 receptors.
PROPEL 2 (NCT04265651) is a phase 2 dose escalation and dose expansion trial and the first clinical trial to study infigratinib at low doses in children with achondroplasia. The goal of the study is to assess safety and measure changes from baseline in annualized height velocity and changes in other health factors. The study is still recruiting at sites across the world, and initial data is expected in the first half of 2022. To learn more about this trial, visit https://www.qedtx.com/achondroplasia/
To learn more about achondroplasia and other rare musculoskeletal disorders, go to checkrare.com/diseases/musculoskeletal