BioMarin initiates Phase III study of vosoritide to treat achondroplasia.

BioMarin Pharmaceutical Inc. announced that the company has initiated a global Phase III study for vosoritide, an analog of C-type Natriuretic Peptide (CNP), in children with achondroplasia, the most common form of dwarfism. The first child enrolled in the study was at a site in Australia.

The Phase III study is a randomized, placebo-controlled study of vosoritide in approximately 110 children with achondroplasia ages 5-14 for 52 weeks. The study will be followed by a subsequent open-label extension. Children in this study will have completed a minimum six-month baseline study to determine their respective baseline growth velocity prior to entering the Phase III study. Vosoritide is being tested in children whose growth plates are still open. This is approximately 25 percent of people with achondroplasia. The primary endpoint of the study is the change in growth velocity from baseline over one year in children treated compared to placebo. The company also plans to augment the growth velocity data with assessments of proportionality and functionality.

In addition, BioMarin is planning a separate Phase II study evaluating the effect of vosoritide in infants and toddlers. Vosoritide has been granted orphan drug designation in both the United States and Europe.