Vosoritide Increases Growth in Children with Achondroplasia

Compared with placebo, a 1-year course of once-daily subcutaneous vosoritide is shown safe and efficacious.

No therapies currently exist to correct endochondrial ossification from achondroplasia. Now, investigators report findings of an industry-sponsored, randomized, placebo-controlled study of vosoritide, a C-type natriuretic peptide, for increasing growth in children with achondroplasia.

Treatment comprised 52 weeks of a once-daily subcutaneous dose of vosoritide 15 µg/kg or placebo in 120 children aged 5–17 years. Results at 1 year were as follows:

Change in annualized growth velocity from baseline was +1.71 cm/year compared with +0.13 cm/year with placebo (P<0.0001).
Change from baseline in height Z-score was +0.27 with vosoritide compared with −0.01 with placebo (P<0.0001).
A serum marker for endochondrial ossification was elevated with vosorit…

Reviewing Author

F. Bruder Stapleton, MD

Disclosures

Consultant/Advisory BoardCareer Physician

Editorial BoardsUpToDate

Leadership Positions in Professional SocietiesAmerican Society of Pediatric Nephrology Foundation (Chair)

F. Bruder Stapleton, MD

Disclosures

Consultant/Advisory BoardCareer Physician

Editorial BoardsUpToDate

Leadership Positions in Professional SocietiesAmerican Society of Pediatric Nephrology Foundation (Chair)

Vosoritide Increases Growth in Children with Achondroplasia

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Reviewing Author