Preliminary data from an open-label, dose-finding Phase 2 clinical trial evaluating BioMarin Pharmaceutical’s (BMRN -1.2%) vosoritide in children with achondroplasia showed a positive effect on height gain. The data were presented at the company’s R&D Day.
Subjects in Cohort 3 (n=10) receiving 15 µg/kg/day experienced cumulative additional height gain of 9.0 cm at month 54 compared to a natural history dataset. 2.2 cm of the increase was observed in the most recent 12 months.
Achondroplasia is a bone growth disorder characterized by dwarfism.
Topline results from a Phase 3 study should be available by year-end.
No comments:
Post a Comment
Note: Only a member of this blog may post a comment.