News from Biomarin
Biomarin updated the description of the currently ongoing study in which 9 sites distributed in US, France, UK and Australia are enrolling children with achondroplasia to gather information about their natural development.
From the original 48 children and adolescents, now the study is planned to enroll aproximately 200, also changing the age span (now 0 to 13.5 yo) of eligible volunteers.
In summary, this study aims to follow children to learn about their natural growth pace and establish patterns, which might be used to compare to the effects on growth expected after exposure to BMN-111. According to Biomarin (here), participation in this study is required to enter the phase 2 study, the first trial using BMN-11 in affected individuals. You can see more details about the clinical development study here: NCT01603095.
Although Biomarin still says in their website that the phase 2 study is (was) planned to start in the end of 2012 or during the first quarter of 2013, it is more likely that it is delayed. Sponsors must publish a description of the study in clinicaltrials.gov when intend to start a study in US and the current list of studies related to achondroplasia (here) does not include an entry for the phase 2 study.
Cronogram delays are common in drug development and sometimes could be related to the need to comply with requests from regulatory agencies such as the FDA. It is possible that the changes made into the design of NCT01603095 are linked to such a discussion with FDA, which could be, in turn, associated to further changes in the phase 2 study design. Discussions with regulatory agencies prior to study beginning are reassuring because it shows us that the study is being due examined by an independent body.