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BioMarin Announces The Lancet Publishes Detailed Vosoritide Phase 3 Data Demonstrating Statistically Significant Increase in Annualized Growth Velocity (AGV) Over 52 Weeks in Children with Achondroplasia

Published: 2020-09-08 13:01:00 ET

Statistically Significant Increases in Height Z Score

Prespecified Subgroup Analysis Demonstrates Consistent AGV and Z Score increases Over Placebo

SAN RAFAEL, Calif., Sept. 8, 2020 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced that The Lancet has published online results from a randomized, double-blind, phase 3, placebo-controlled, multicenter trial for vosoritide, an investigational analog of C-type Natriuretic Peptide (CNP), in children aged 5 to 18 years with achondroplasia. Achondroplasia is the most common form of disproportionate short stature in humans. The data demonstrated 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.

"This study provides the first robust evidence for a precision therapy for achondroplasia that has the potential to fundamentally change the clinical management, growth trajectory, and treatment recommendations for affected children." said Ravi Savarirayan, M.B., B.S., M.D., lead author of The Lancet study and investigator from the Murdoch Children's Research Institute, Royal Children's Hospital, University of Melbourne, Parkville, Victoria, Australia. "As a treating physician, the lack of therapeutic options for children with achondroplasia represents an unmet medical need in this area."

The primary endpoint was change from baseline in AGV at 52 weeks in participants administered daily subcutaneous injections of vosoritide, at a dose of 15.0 µg/kg/day, compared with placebo. The findings demonstrated that the adjusted mean difference in AGV between children in the vosoritide group and placebo group was 1.57cm per year in favor of vosoritide (95% CI: 1.22 - 1.93, p value

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All statements made via social media sites sponsored or maintained by Enhanced Investments and its affiliates are for informational purposes only and do not constitute a comprehensive description of Enhanced Investments' investment advisory services.
Certain investments are not suitable for all investors. Before investing, consider your investment objectives and applicable fees. The rate of return on investments can vary widely over time, especially for long term investments. Investment losses are possible, including the potential loss of all amounts invested. Information provided by Enhanced Investments is for informational and general educational purposes only and is not investment or financial advice.