Vosoritide for achondroplasia:
In June, the Company published results from the Phase 2 study showing a 50 percent or 2.01 cm/year increase in mean annualized growth velocity (speed at which growth in children occurs) in the cohort of 10 patients receiving a 15 µg/kg dose of vosoritide daily for six months compared with their own pre-treatment growth velocity (p-value= 0.01). Based on these results, BioMarin intends to move into Phase 3 study discussions on the next stage of development with health authorities with a dose of 15 micrograms per kilogram daily. In addition, to support further exploration of a dose that may enable “catch-up” growth in the event of delayed treatment, a fourth cohort with 30 micrograms per kilogram daily has recently completed enrollment in the Phase 2 study. BioMarin will provide an update on Phase 3 design and plans, as well as 12 month results from the Phase 2 study at the R&D Day in April 2016.