BioMarin Pharmaceutical initiates phase 3 vosoritide trial | Courtesy of Shutterstock
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Jeff Gantt | Dec 18, 2016

BioMarin Pharmaceutical initiates phase 3 vosoritide trial

BioMarin Pharmaceutical recently announced that the first steps toward initiating a phase 3 trial involving vosoritide have been taken.

Vosoritide is an equivalent to C-type Natriuretic Peptide (CNP). The studies will focus on vosoritide in children with achondroplasia, otherwise known as the common form of dwarfism.

The first child has been enrolled in the study at a site in Australia.

"Based on the positive results seen in our phase 2 study, we look forward to continuing to investigate vosoritide," BioMarin President of Worldwide Research and Development Dr. Hank Fuchs said. "We are grateful to the children -- and their families -- who have participated in the earlier study and are participating in this phase 3 study."

This phase 3 trial will enroll approximately 110 children between 5 and 14 years old who have achondroplasia over a 52-week span.

The trial will be randomized, placebo-controlled and followed by an open-label extension. Candidates for the study are required to have completed a minimum six-month baseline study that is designed to determine their baseline growth velocity before they can be enrolled in the trial.

Vosoritide is only being administered to children who still have open growth plates.

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