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BioMarin initiates phase 3 trial of vosoritide for achondroplasia in children

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BioMarin initiates phase 3 trial of vosoritide for achondroplasia in children

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.