Vosoritide Phase III study shows significant increase in annualized growth velocity in achondroplasia

California, USA-base BioMarin Pharmaceutical (Nasdaq: BMRN) today announced that The Lancet has published online results from a randomized, double-blind, Phase III, placebo-controlled, multicenter trial for vosoritide, an investigational analog of C-type natriuretic peptide (CNP), in children aged 5 to 18 years with achondroplasia.

Achondroplasia is the most common form of disproportionate short stature in humans. The data demonstrated that daily subcutaneous administration of vosoritide to children with achondroplasia resulted in significantly increased growth velocity and height Z scores over baseline after one year of treatment as compared to those who received placebo with similar adverse effect profiles.

"This study provides the first robust evidence for a precision therapy for achondroplasia that has the potential to fundamentally change the clinical management, growth trajectory, and treatment recommendations for affected children," said Dr Ravi Savarirayan, lead author of The Lancet study and investigator from the Murdoch Children's Research Institute, Royal Children's Hospital, University of Melbourne, Parkville, Victoria, Australia. "As a treating physician, the lack of therapeutic options for children with achondroplasia represents an unmet medical need in this area," he noted.