Biomarin Presents on Achondroplasia 2-Year Study Results

 BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced that data from the open-label long-term extension of the Phase 3 study of 15 µg/kg dose of vosoritide was presented at an oral presentation at ENDO21, the Endocrine Society's Annual Meeting by Professor Ravi Savarirayan, M.B., B.S., M.D., clinical investigator from the Murdoch Children's Research Institute, Royal Children's Hospital, University of Melbourne, Parkville, Victoria. Vosoritide is an investigational, once daily injection analog of C-type Natriuretic Peptide (CNP) for the treatment of achondroplasia, the most common form of disproportionate short stature in humans. 

The data from the open-label extension presented at ENDO21 showed that children maintained an increase in Annual Growth Velocity (AGV) through the second year of continuous treatment with vosoritide.  Children who received two years of vosoritide therapy had a baseline mean AGV of 4.28 cm/year. After one year of treatment, mean AGV was 5.71 cm/year and after the second year mean AGV was 5.65 cm/year, demonstrating sustained restoration of a major portion of the growth deficit in achondroplasia through the second year of treatment. Children also had an improved height z-score, which is a measure of height relative to that of a similar population of average height.

https://www.prnewswire.com/news-releases/biomarin-announces-oral-presentation-at-endo2021-the-endocrine-societys-annual-meeting-with-data-demonstrating-2-years-of-treatment-benefit-in-children-with-achondroplasia-treated-with-vosoritide-301252378.html