Achondroplasia Treatment

Achondroplasia is a genetic condition caused by a gene alteration in the fibroblast growth factor receptor (FGFR3). The protein begins to function abnormally, slowing down the growth of bone in the cartilage of the growth plate.  People with achondroplasia have short stature with short arms, legs and fingers and may also have breathing problems, including apnea. Other health issues include obesity and recurrent ear infections. Adults with achondroplasia may develop sway of the lower back and bowed legs, leading to lower back pain and difficulty with walking.

Current treatments include daily subcutaneous injection of vosoritide, a C-natriuretic peptide analog, recently approved to improve growth velocity in children with achondroplasia. 

The goal of Ferdia’s program is to develop a safe and effective, pre-formulated alternative suitable for once-weekly treatment.