An update on the BioMarin MPS IIIB clinical development programme

An update on the BioMarin MPS IIIB clinical development programme

An update for patient associations and families from BioMarin
April 2017

BioMarin have received a number of enquiries regarding the status of their clinical development program in Sanfilippo Syndrome Type B (MPS IIIB). The program, which involves multiple centres around the world, remains open and is actively enrolling. The program is enrolling up to 30 children between the ages of 1 and 10 and consists of two studies, described below.

The first study is an observational study of children with Sanfilippo Type B and includes testing of cognitive and adaptive function. The observational study lasts for 48 weeks. These 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 behaviour and quality of life. This study is intended to provide baseline information about how Sanfilippo Type B progresses in the absence of treatment. This baseline information can then be compared to disease progression information from BioMarin’s subsequent treatment study (both for individual children and in aggregate).

The observational study is enrolling at sites in Australia, Colombia, Germany, Spain, Taiwan, Turkey, UK and USA.

First study information

The second study is a treatment study in which children with Sanfilippo Type 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. This study will be run at centres which are also part of the observational study. To enroll in the second phase of the treatment study, a child must have completed the observational study as outlined above, or have completed the first part of the treatment study.

Second study information

Any questions or concerns you may have should be directed to your child’s doctor, who remains the best source of information about the care of your child. For more information about active clinical studies, including Sanfilippo Type B, please visit

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