BioMarin are pausing further recruitment for their BMN-701 clinical trial and have provided this programme update:

January 2015

BioMarin is pleased to update the Pompe patient community on the progress made in our programme to develop a treatment for people living with Pompe disease.

Over the past year, BioMarin has initiated a global clinical development program of BMN-701 treatment in Pompe patients this is currently ongoing in over 20 countries. The program includes three studies: a switchover study in patients currently treated with commercially available enzyme replacement therapy (ERT), an observational study in Pompe patients treated or untreated with commercially available ERT, and a study to better understand efficacy endpoints used in clinical trials.

We have decided to move to an updated manufacturing process. This has no reflection on the safety or efficacy of the drug but we believe it will provide better long term availability. In order to avoid having to repeat the switchover study to generate the necessary data for regulatory filings, we are pausing enrolment of the switchover study so that the revised material can be introduced. Enrolment to the study will recommence when new material is ready in the second half of 2015.

Patients currently enrolled and receiving BMN 701 study drug will not be affected by this change and will continue to receive existing study drug. They will have the opportunity to continue treatment in the study with the revised drug once it is available. There are no changes planned to the observational or the endpoint studies and enrollment will continue throughout this period.

If you have queries or concerns about your enrolment or participation in this study please contact your treating clinician or medical team.

BioMarin is committed to the Pompe community and to developing a therapy for late onset Pompe disease. We thank you for your ongoing commitment and involvement in this study.