(RTTNews) - BioMarin Pharmaceutical Inc. (BMRN) said data from a randomized, double-blind, phase 3, placebo-controlled, multicenter trial for vosoritide showed that daily subcutaneous administration of vosoritide to children with achondroplasia resulted in significantly increased growth velocity and height Z scores over baseline after one year of treatment as compared to those who received placebo with similar adverse effect profiles. Vosoritide was generally well tolerated in the study.
BioMarin said an ongoing, open-label, phase 3, extension study will continue to evaluate the balance of benefits and harms of vosoritide until the children reported in the study reach final adult height.
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