BioMarin: Vosoritide Phase 3 Study Shows Positive Results - Quick Facts

(RTTNews) - BioMarin Pharmaceutical Inc. (BMRN) reported positive final results from its double-blind, placebo-controlled Phase 3 study evaluating the efficacy and safety of vosoritide in Children with achondroplasia. In the study, placebo-adjusted increase in growth velocity was 1.6 cm/yr. Vosoritide was generally well tolerated.

Based on the results, the company plans to meet with health authorities in the first half of 2020 to discuss plans for submitting marketing applications.

"The placebo-controlled study demonstrated a strong increase in growth velocity across the broad population studied. These results when combined with the long-term benefits seen in the phase 2 study provide hope for a significant and sustained benefit for children with achondroplasia," said Hank Fuchs, President Worldwide Research and Development at BioMarin.

Shares of BioMarin Pharma were up 7% in pre-market trade on Monday.