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

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 (ClinicalTrials.gov 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.

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