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# Biomarin’s Short Stature Drug Accelerated Approval
- URL: https://www.fdaweb.com/biomarins-short-stature-drug-accelerated-approval/
- Published: 2021-11-19T12:00:00.000Z
- Updated: 2026-09-14T17:22:57.000Z
- Author: David McFarland
- Tags: Biologics, #legacy-id-D5150618

FDA has granted BioMarin accelerated approval for Voxzogo (vosoritide) for injection, indicated to increase linear growth in pediatric patients with achondroplasia, the most common form of disproportionate short stature, in those five years of age and older with open epiphyses (growth plates). The approval was based on data showing an improvement in annualized growth velocity. To fulfill a confirmatory study post-marketing requirement, BioMarin says it will use ongoing open-label extension studies compared to available natural history.

Voxzogo is described by the company as a C-type natriuretic peptide analog, which acts as a positive regulator of the signaling pathway downstream of FGFR3 to promote endochondral bone growth. Its approval also came with a Rare Pediatric Disease Priority Review Voucher, which permits a priority review to a subsequent drug application that would not otherwise qualify for priority review.