The most relevant change in the information posted is the inclusion of an extension of 24 months to the 6 month interventional study. Given that the study was designed to check its safety (primary outcome), this could be interpreted as a sign that the drug is not causing harm in the short term. Furthermore, as the study was designed also to capture efficacy (secondary outcome), what is called "proof of concept", one could also understand the extension of the study as a positive indication for this parameter, too.
Why is that so? Because an experimental drug needs to prove its safety and efficacy in all phases of the clinical development. The developer will only progress the research of a new medicine if there is enough evidence establishing that success on these two key parameters are being achieved in each phase.
In public meetings (*), Biomarin has been declaring that the results of the study will be divulged by the end of the second trimester of 2015. Therefore, we will have to wait for the release of the complete results of the study before making assumptions. Sometimes, early signals of positive results are not confirmed when the complete study data is analysed. For instance, the drug shows safety, but the improvement seen in the condition studied is not significant enough to grant further development.
Biomarin has also updated the information about the natural history study, changing the study status from "active, not recruiting" (August 2014) to "active, enrolling by invitation", which could mean that they are planning to increase the number of participants in the BMN-111 development plan. This seems to be a positive information, too, but the developer has not disclosed any clear news about this yet.
Here are the links for the study registries at clinicaltrials.gov:
- A Phase 2 Study of BMN 111 to Evaluate Safety, Tolerability, and Efficacy in Children With Achondroplasia
* To follow Biomarin public releases, visit their website here.
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