BioMarin Pharmaceutical Inc. initiated a global phase 3 study of vosoritide for the treatment of children with achondroplasia, the most common form of dwarfism.
The randomized and placebo-controlled phase 3 study involves about 110 children aged 5 to 14 with achondroplasia and will run for 52 weeks. The study's primary endpoint is the change in growth velocity from baseline over one year compared to placebo, BioMarin said in a news release.
BioMarin is also planning a separate phase 2 study to evaluate the effect of vosoritide in infants and toddlers. Vosoritide is an analog of C-type natriuretic peptide.