The New Drug Application (NDA) for vosoritide (BioMarin), an investigational analogue of C-type natriuretic peptide, has been submitted to the Food and Drug Administration (FDA) for the treatment of children with achondroplasia, a genetic disorder that results in disproportionate short stature and significant health complications. 

The NDA submission includes data from a double-blind, placebo-controlled, phase 3 study assessing vosoritide in 121 children aged 5 to 14 years with documented achondroplasia confirmed by genetic testing. Patients were randomized to receive once daily subcutaneous injections of vosoritide or placebo over 1 year. The primary end point was the change from baseline in mean annualized growth velocity.

Findings from the study demonstrated that patients treated with vosoritide achieved a placebo-adjusted increase from baseline in growth velocity of 1.6cm/yr (P <.0001); the results were reported to be consistent across a broad patient population. Vosoritide was found to be generally well tolerated; injection site reactions were reported as the most common treatment-emergent adverse events.

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“This regulatory submission is an important scientific and medical milestone for children with achondroplasia and their families,” said Julie Hoover-Fong, MD, PhD, FACMG, Professor, McKusick-Nathans Department of Genetic Medicine and Director, Greenberg Center for Skeletal Dysplasias at Johns Hopkins University and an investigator in the vosoritide clinical program.  “The extensive clinical program for vosoritide includes important natural history information, which has contributed to the body of scientific knowledge about achondroplasia and a potential treatment option for patients.”

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  1. BioMarin submits new drug application to US Food and Drug Administration for vosoritide to treat children with achondroplasia. Accessed August 20, 2020. 
  2. BioMarin announces positive final results from placebo-controlled phase 3 data in children with achondroplasia treated with vosoritide. Accessed August 20, 2020.