BioMarin Seeks to Expand Use of Vosoritide to Younger Children With Achondroplasia

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The Food and Drug Administration (FDA) has accepted the supplemental New Drug Application (sNDA) for vosoritide for the treatment of children less than 5 years of age with achondroplasia, a genetic disorder that results in disproportionate short stature and significant health complications.

The sNDA is supported by data from a double-blind, placebo-controlled phase 2 trial ( Identifier: NCT03583697) that evaluated the efficacy and safety of vosoritide in patients less than 5 years of age with achondroplasia. Patients were randomly assigned to receive either vosoritide subcutaneously once daily (n=43) or placebo (n=32). The primary endpoint was the change from baseline in height Z-scores at 52 weeks.

Findings showed that treatment with vosoritide increased height Z-score by 0.30 (95% CI, 0.07-0.54) and annualized growth velocity of 0.92cm/year (95% CI, 0.24-1.59). Improvement in height Z-score was found to be consistent with that seen after 1 year of treatment in patients over the age of 5. Compared with placebo, no significant treatment effect was observed on upper-to-lower body segment ratio with vosoritide.

“We are pleased that the FDA has accepted our sNDA and are working closely with the agency to facilitate completion of the review in a timely manner,” said Hank Fuchs, MD, president, Worldwide Research and Development at BioMarin. “There are currently no approved pharmacological treatments in the United States for children under 5 with achondroplasia and this approval could potentially extend access to all children with achondroplasia, whose growth plates are not closed.”

A Prescription Drug User Fee Act target date of October 21, 2023 has been set for the application.

Vosoritide, an analogue of C-type natriuretic peptide, is currently marketed under the brand name Voxzogo to increase linear growth in children 5 years of age and older with achondroplasia and open epiphyses.


  1. FDA accepts BioMarin’s supplemental New Drug Application to expand use of Voxzogo® (vosoritide) for injection to treat children with achondroplasia under the age of 5. News release. BioMarin Pharmaceutical. Accessed March 8, 2023.
  2. BioMarin announces favorable results from global phase 2 study of Voxzogo (vosoritide) for injection in infants and young children with achondroplasia at The Endocrine Society Annual Meeting, ENDO 2022 (June 11-14), in Atlanta. News release. BioMarin Pharmaceutical. June 13, 2022. Accessed March 8, 2023.,-ENDO-2022-June-11-14-,-in-Atlanta.