Thursday, July 30, 2015

Treating achondroplasia: news from the vosoritide clinical development program

Biomarin has just updated the registry of the study they are conducting to assess the natural growth patterns of children with achondroplasia. Participation in this clinical assessment study is required, according with the sponsor for a potential inclusion in the phase 2 vosoritide study, according with the information posted. The information collected in this study is used to compare with the growth patterns after the initiation of the therapy in the drug study (2).

It is noteworthy that the list of participating investigators has not been increased yet, although this could be expected in some moment in the near future, if we think that the phase 3 study design will keep using the natural growth pattern as the comparator for efficacy of the drug, in the same way used in the phase 2.

Here is the link for


1. Biomarin press release. 17 Jun 2015.

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