BioMarin doses first participant in Phase 2 study of achondroplasia candidate
BioMarin dosed the first participant in a global Phase 2 study for vosoritide, an analog of C-type Natriuretic Peptide, in infants and young children with achondroplasia, the most common form of disproportionate short stature in humans. The Phase 2 study is a study of vosoritide in approximately 70 infants and young children with achondroplasia ages zero to less than 60 months for 52 weeks. The study will be followed by a subsequent open-label extension. The primary objectives of the study are to evaluate safety, tolerability, and the effect of vosoritide on height Z-scores, which is the number of standard deviations in relation to the mean height of age-matched, average stature children. Vosoritide has been granted orphan drug designation in both the United States and Europe.