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Clinical Trials

Date: 2016-12-12

Type of information: Recruitment of the first patient

phase: 3

Announcement: recruitment of the first patient

Company: Biomarin Pharmaceutical (USA - CA)

Product: BMN 111 (vosoritide)

Action mechanism:

peptide/natriuretic peptide. Vosoritide is an analog of C-type Natriuretic Peptide (CNP). Vosoritide has Orphan designation in both the United States and Europe.

Disease: achondroplasia

Therapeutic area: Rare diseases - Genetic diseases

Country: Australia

Trial details:

Latest news:

* On December 12, 2016, BioMarin Pharmaceutical announced 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. 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.

Is general: Yes