Additional Relevant Details BioMarin Pharmaceutical 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. After 12 months of daily dosing at 15 µg/kg/day, the cohort 3 patients (n=10) experienced a 46% or 1.9 cm/year increase in mean annualized growth velocity from baseline (p-value = 0.02). (See Table 2). These findings provide evidence of durability of effect consistent with previously presented 6-month data for these patients, which demonstrated an annualized increase of 50% or 2.0 cm/year in mean annualized growth velocity. In addition, 6-month data for 12 patients who were initiated on a lower dose and switched to 15 µg/kg/day showed an increase of 65% or 2.3 cm/year in mean annualized growth velocity from baseline (p-value = 0.002). Vosoritide at 15 µg/kg/day was well tolerated and there were no treatment-related serious adverse events or adverse events leading to discontinuation. Consistent with the initial 6-month data, in the newly released data, all adverse events assessed as related to study drug were mild to moderate. Over 12 months of dosing at 15 µg/kg/day, injection site reactions and hypotension (asymptomatic decreases in blood pressure) were the most common drug related adverse events, reported by 90% and 40% of cohort 3 patients, respectively. All injection site reaction events were mild and the majority were resolved in one hour. All adverse events of hypotension were mild, transient and resolved without intervention. Safety data from lower dose cohort subjects who increased their dose to 15 µg/kg/day for at least 6 months was generally comparable to the 12-month safety data; however, two subjects experienced symptomatic events associated with a decrease in blood pressure, only one of which was deemed by the investigator to be related to study drug. Both subjects continued to receive their daily dose of vosoritide without dose modification. The phase 2 study is also evaluating a higher dose of 30 ug/kg/day. Preliminary data after several months of treatment has shown that this dose is similarly well tolerated with no new safety findings.