Research & Development
BioMarin Pharmaceutical receives US FDA accelerated approval for VOXZOGO for injection
22 November 2021 -

BioMarin Pharmaceutical Inc (NASDAQ: BMRN), a US-based global biotechnology company, announced on Friday that it has received accelerated approval from the United States Food and Drug Administration (FDA) for its VOXZOGO (vosoritide) for Injection intended to raise linear growth in paediatric patients with achondroplasia five years of age and older with open epiphyses (growth plates).

The indication has been approved under accelerated approval based on an improvement in annualised growth velocity. Continued approval for this indication is likely to be contingent upon verification and description of clinical benefit in confirmatory studies. In order to complete this post-marketing requirement, the company aims to use the ongoing open-label extension studies compared to available natural history.

The product is the first US FDA approved treatment for children with achondroplasia. It is likely to be offered in the United States by mid- to late-December, and BioMarin is to commence promotion of the product immediately. The approval for the product was based on the results of a global randomised, double-blind, placebo-controlled Phase three study assessing the efficiency and safety of the product and the open-label extension of this Phase three study.