BioMarin starts the phase 2 study BMN 111-206 in infants and toddlers

On the 18th June 2018, it was made public that the first patient under 5 years-old was dosed with Vosoritide.

The BMN 111–206: Phase 2 Infant and Toddler Study will evaluate the effect of BMN 111 in approximately 70 infants and toddlers between the ages of 0 to 5.

This is a randomized, placebo-controlled, double-blind multicentre study. This means that participants have an equal chance of receiving either placebo (an inactive treatment that looks the same as, and is given in the same way as, the investigational therapy) or the investigational product BMN 111. It is not known whether placebo or investigational therapy is being administered to prevent bias (errors).

This trial is opening in Australia, Japan, the United Kingdom and the United States and participants must remain a resident of the country they enrolled in throughout the trial period. 

Recently, in an FDA public hearing for achondroplasia (you can read more here) that was oriented to discuss the value and the design of clinical trials for achondroplasia, one of the most important topics was the need to start clinical trials in younger children, once the typical complications due to achondroplasia tend to occur under 2 years-old.

As ALPE stated in this meeting "Timing is a clue aspect. Given the worsening of the associated risks to achondroplasia, as time goes by, families with their children can get a good life quality as soon as possible. They are afraid trials go on forever." And also "Another aspect we worry about is the possibility or not that children under five are able to start the potential drug. This is a race against time: the sooner we start, the higher the benefits. We don’t want our children to miss five years of potential improvement".

So this new study is a manifest of collaboration between regulators: EMA and FDA, pharmaceutical companies and patient organizations.

You can read the full press release here.