BioMarin Provides Encouraging Preliminary Data on First 8 Patients in Hemophilia A Gene Therapy Program

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BioMarin Pharma (BMRN) Updates on Vosoritide Phase 2 in Achondroplasia; Evidence of Durability Noted

April 20, 2016 10:40 AM EDT

BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) 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... More