Update on the BioMarin Sanfilippo Syndrome Type B (MPS IIIB) Clinical Development Program

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Update on the BioMarin Sanfilippo Syndrome Type B (MPS IIIB) Clinical Development Program

An Update for Patient Associations and Families from BioMarin
May 2018

We would like to update you about the status of our clinical development program in Sanfilippo B. The BioMarin program, which involves multiple centers around the world, consists of three studies, described below.

The first study is an observational study of children with Sanfilippo B and includes testing of cognitive and adaptive function. The observational study lasts for 48 weeks. The tests explore how the child thinks and acquires new information as well as how the child deals with daily living. There are also assessments of behavior and quality of life. This study is intended to provide baseline information about how children with Sanfilippo B progress in the absence of treatment. This baseline information may then be compared to disease progression information from BioMarin’s treatment study (both for individual children and in aggregate).

The observational study is being conducted at sites in Australia, Colombia, Germany, Spain, Taiwan, Turkey, UK and USA. Enrollment to this study is now complete.

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The second study is a treatment study in which children with Sanfilippo B will receive an investigational enzyme replacement therapy, known as BMN 250. The enzyme is administered directly to the brain as an infusion via a surgically implanted port. To enroll in the treatment study, a child must have completed the observational study as outlined above. This study is being conducted at the same centers. Children who have previously received an investigational drug as part of another clinical study, including gene therapy, may not be enrolled in this study.

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The third study is a natural history study of children with Sanfilippo B up to 18 years old, who do not meet the criteria for participation in the observational study. Children enrolled into this study do not receive the investigational treatment. The objective of this study is to better understand the natural course of Sanfilippo B.

More details

Your child’s doctor remains the best source of information regarding the care of your child and any questions or concerns should be directed to your child’s doctor.

If you represent a patient association, please contact BioMarin Patient Advocacy patientadvocacy@bmrn.com. Alternatively, please email BioMarin Medical Information medinfo@bmrn.com

For more information about active clinical studies, including Sanfilippo Type B, please visit www.clinicaltrials.gov or www.clinicaltrialsregister.eu