Treatment with vosoritide (Biomarin),  an investigational analog of C-type natriuretic peptide, was found to increase growth velocity in children with achondroplasia, a genetic disorder that results in disproportionate short stature and significant health complications.

The placebo-controlled, double-blind, phase 3 study included 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 endpoint of the study was change from baseline in mean annualized growth velocity. 

Results showed a placebo-adjusted increase from baseline in growth velocity of 1.6cm/yr (P <.0001) in children treated with vosoritide; the findings were reported to be consistent across a broad patient population. With regard to safety, most adverse events were considered mild with no clinically significant decreases in blood pressure; injection site reactions were the most common treatment-emergent adverse events.

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“These results when combined with the long-term benefits seen in the phase 2 study provide hope for a significant and sustained benefit for children with achondroplasia,” said Hank Fuchs, MD, President of R&D at Biomarin. “We look forward to discussing plans for submitting marketing applications with health authorities.”


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