BioMarin Pharmaceutical (BMRN +3.3%) announces positive results from a Phase 3 clinical trial evaluating vosoritide (BMN 111) in children with achondroplasia, a bone growth disorder characterized by dwarfism.
The study met the primary endpoint of a statistically significant change from baseline in mean annualized growth velocity at week 52 compared to placebo.
The company plans to meet with regulatory authorities in H1 2020 to clarify plans for marketing applications.
Vosoritide is a stabilized version of C-type natriuretic peptide, a natural human peptide that is a positive regulator of bone growth.
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