As we have previously reported, the Phase III trial for Batten-1 is experiencing some unanticipated delays due to funding issues. However, the program continues to be the highest priority for BBDF and Theranexus. Together, we are working hard to secure the significant financial resources necessary to see this program through to potential regulatory approval.
The delay in the Phase III portion of the Batten-1 clinical trial is providing an opportunity for us to further review the FDA’s feedback on the study design and discuss and consider alternative regulatory strategies that will seek to bring the drug to patients within the shortest possible time frame. To this end, we are working with Engage Health to collect information from patients who are currently using off-label miglustat in consultation with their medical practitioner. Please note that taking miglustat off-label will not exclude a patient from participating in a future trial, and this data may be helpful in informing our efforts and potentially revising the study design.
If your child is currently using off-label miglustat in consultation with your medical practitioner, we would be so grateful for your participation.
If you know of other families using miglustat off-label, please share this information with them.
We are anxious to get started on Phase lll. If you have any questions, please don’t hesitate to reach out to me.
Many thanks,
Mary Beth Kiser
President and CEO