Highest Dose (30 µg/kg/day) Shows Approximately 50% Increase in Mean Annualized Growth Velocity, Comparable with 15 µg/kg/day dose
Consistent Safety Profile at High Dose
Findings Support 15 µg/kg/day in Phase 3, Randomized Controlled Study to Start by End of 2016
SAN RAFAEL, Calif., Oct. 19, 2016 (GLOBE NEWSWIRE) -- BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) today provided an update on its Phase 2 study of vosoritide, an analog of C-type Natriuretic Peptide (CNP), 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 µg/kg/daily experienced a 46% or 2.1 cm/year increase in mean annualized growth velocity from baseline (p-value = 0.03). These data are comparable to those observed at the lower dose of 15 µg/kg/day in cohort 3. Results from 10 children in cohort 3, who completed six months of daily dosing at 15 µg/kg/day experienced a 50% or 2.0 cm/year increase in mean annualized growth velocity from baseline (p-value = 0.01). (See Table 2.)
Vosoritide was generally well tolerated at all doses. The majority of adverse events (AEs) were mild and no serious AEs were reported as study drug-related. Across all doses, injection site reactions and hypotension were the most common drug-related AEs. All injection site reaction events were mild and transient. AEs of hypotension were mild, transient and resolved without medical intervention, and the majority were asymptomatic and reported in context of routine blood pressure measurements. No new safety findings were observed at the 30 µg/kg/day dose.
“Studying the higher dose in Phase 2 informed the design of our Phase 3 study in vosoritide. While vosoritide at the higher dose of 30 µg/kg/day was generally well-tolerated, the data support our use of the lower dose of 15 µg/kg/day in the Phase 3 study,” said Hank Fuchs, MD, Chief Medical Officer at BioMarin. “We believe that growth velocity is an important measurement in developing vosoritide, which has the potential to address the complications associated with achondroplasia. We are grateful to the children and their families who are participating in this study.”
“Vosoritide represents a potential, first-of-its-kind treatment for this form of dwarfism, and these clinical studies could provide new insights into improved management of this condition,” said Julie Hoover-Fong Director, Greenberg Center for Skeletal Dysplasias, Johns Hopkins University and lead author of the poster of the Vosoritide Phase 2 data update at ASHG.
For full article, please click here.
