Press release on Vosoritide

The latest press release about vosoritide was presented in January 2016 by Jean-Jacques Bienaimé, the CEO of BioMarin. The number of children BioMarin estimates that can be treated by vosoritide is around 24.000 children worldwide.

This number reflects only children with achondroplasia with open growth plates, once the estimated number for the global population with achondroplasia is around 95.500 people.

The relevant points presented on this product under clinical trial for achondroplasia were BioMarin has received approval by the health authorities to go forward with vosoritide worldwide and that vosoritide will proceed to phase 3.



In April 2016, BioMarin will share the plans for phase 3 and also the results of the 1 year data with the 15 μg/kg dose.

Vosoritide in now in phase 2, with 26 children participating in the drug clinical trial. Most probably, the shared plans will be the new clinical centres where phase 3 will be run and the inclusion and exclusion criteria, that are absolutely necessary to determin which children are elegible to be recruted for phase 3.

This phase is predicted to have one year duration.

BioMarin predicts to start Phase 3 in the 4th quarter of 2016, somewhere between October and December 2016.

Another new point revelead is that children under 5 years old will be included in a study to explore earlier treatment option.