Monday, December 12, 2016

Treating Achondroplasia: Phase 3 study with vosoritide started in Australia.

Biomarin, the developer of vosoritide (BMN-111), the first potential therapy for achondroplasia to reach clinical development, has just announced that the first child has been enrolled in the phase 3 study, in Australia.

 This is the press release:

BioMarin Enrolls First Participant in Phase 3 Trial of Vosoritide for Treatment of Children with Achondroplasia

SAN RAFAEL, Calif., Dec. 12, 2016 (GLOBE NEWSWIRE) -- BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) announced today that the company has initiated a global Phase 3 study for vosoritide, an analog of C-type Natriuretic Peptide (CNP), in children with achondroplasia, the most common form of dwarfism. The first child enrolled in the study was at a site in Australia.
"Based on the positive results seen in our Phase 2 study, we look forward to continuing to investigate vosoritide," said Hank Fuchs, M.D., President, Worldwide Research and Development at BioMarin. "We are grateful to the children and their families, who have participated in the earlier study and are participating in this Phase 3 study."
The Phase 3 study is a randomized, placebo-controlled study of vosoritide in approximately 110 children with achondroplasia ages 5-14 for 52 weeks. The study will be followed by a subsequent open-label extension. Children in this study will have completed a minimum six-month baseline study to determine their respective baseline growth velocity prior to entering the Phase 3 study. Vosoritide is being tested in children whose growth plates are still open. This is approximately 25 percent of people with achondroplasia.
The primary endpoint of the study is the change in growth velocity from baseline over one year in children treated compared to placebo.  The company also plans to augment the growth velocity data with assessments of proportionality and functionality.
In addition, BioMarin is planning a separate Phase 2 study evaluating the effect of vosoritide in infants and toddlers. Vosoritide has been granted orphan drug designation in both the United States and Europe.

Here goes a link for the complete press release:

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