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BioMarin Announces New England Journal of Medicine Publishes Vosoritide Phase 2 Study Showing Sustained Annualized Growth Up to 42 Months in Children with Achondroplasia
SAN RAFAEL, Calif., June 18, 2019 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) announced that the New England Journal of Medicine (NEJM)
About this update from Biomarin Pharmaceutical Inc.
[{"type":"text","content":"SAN RAFAEL, Calif., June 18, 2019 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) announced that the New England Journal of Medicine (NEJM) published online today results from a Phase 2 dose-finding and extension study for vosoritide, an investigational analog of C-type Natriuretic Peptide (CNP), in children with achondroplasia. The data demonstrated that vosoritide was generally well tolerated with a mild side effect profile and resulted in a sustained increase in annualized growth velocity for up to 42 months in children aged 5 to14 years with achondroplasia, the most common form of disproportionate short stature in humans. The results will also appear in the July 4th printed issue.\n\n \nIn addition, the company announced that it had met its enrollment goal of the first cohort (n=30) of a separate Phase 2 study of vosoritide in infants and young children ages two to five years.\n\"Our research continues to investigate the potential of vosoritide to assist in skeletal growth in children with achondroplasia,\" said Ravi Savarirayan, M.B., B.S., M.D., lead author of the NEJM study and investigator from the Murdoch Children's Research Institute, Royal Children's Hospital, University of Melbourne, Parkville, Victoria, Australia. \"I am hopeful that this ongoing clinical development program will be able to demonstrate a meaningful difference for children with achondroplasia.\"\nThe NEJM publication described results of an ongoing open-label, Phase 2 study in children with achondroplasia, where vosoritide demonstrated a sustained increase in height and associated height Z scores for up to 42 months of treatment in children in cohort 3 receiving a continuous dose of 15 µg/kg/day. Annualized growth velocity increased from baseline in all cohorts during each 12-month interval by 1.10 to 2.34 cm/year through 42 months. In cohort 3 (n=10) receiving 15 μg/kg continuous dosing from baseline, the mean annualized growth velocity derived between 30 and 42 months was 5.51 cm/year representing a 1.46 cm/year (95% CI −0.15, 3.07) change from baseline. In cohort 4 (n=9) receiving 30 μg/kg continuous dosing from baseline, the mean annualized growth velocity between 18 and 30 months was 5.60 cm/year representing a 1.10 cm/year (95% CI −0.27, 2.48) change from baseline.\n\"The results published today by NEJM demonstrate the importance of ...