BioMarin reports Phase 2 vosoritide data in dwarfism
BioMarin provided an update on its Phase 2 study of vosoritide in children with achondroplasia, the most common form of dwarfism, at the American Society of Human Genetics 2016 Meeting. Results from 8 children in cohort 4, who completed six months of daily dosing at 30 microgram/kg/daily experienced a 46% or 2.1 cm/year increase in mean annualized growth velocity from baseline. These data are comparable to those observed at the lower dose of 15 microg/kg/day in cohort 3, the company said. Results from 10 children in cohort 3, who completed six months of daily dosing at 15 microgram/kg/day experienced a 50% or 2.0 cm/year increase in mean annualized growth velocity from baseline. Vosoritide was generally well tolerated at all doses. All injection site reaction events were mild and transient. No new safety findings were observed at the 30 microg/kg/day dose. "The data support our use of the lower dose of 15 microgram/kg/day in the Phase 3 study," the company said, adding that "the company believes based on discussions with global health authorities that change in growth velocity from baseline as an endpoint could lead to registration. The company plans to augment these data with supportive evidence concerning proportionality and functionality."