BioMarin reports positive early-stage results for trial drug to treat CLN2 disease
pharmafile | March 4, 2016 | News story | Medical Communications |
Biotechnology firm BioMarin Pharmaceutical has announced positive results from early stage trial for drug to treat neurodegenerative disease in children.
Based on the study data, the company plans to file for US and European marketing applications in the middle of the year. Following the regulatory nod, the drug, cerliponase alfa, will become the first effective treatment for CLN2 disease, a form of Batten disease.
Hank Fuchs, chief medical officer of BioMarin, said, “Maintaining one or two points on the CLN2 disease-specific rating scale could mean the important difference between a child being able to continue to walk and talk or not. We have pushed out a medical frontier by developing a potential first enzyme replacement therapy administered directly into the brain ventricles for a form of Batten disease. We are planning to apply for regulatory approval in the US and Europe with this study, which is a very rapid registration approach justified by compelling data in this devastating disease.”
CLN2 disease, a form of Batten disease, is a rapidly progressing, fatal neurodegenerative disease with no approved treatments, where the majority of affected children lose their ability to walk and talk by about six years of age.
Angela Schulz, department of paediatrics, University Medical Center Hamburg-Eppendorf, said, “We have seen a highly significant impact for the children participating in this study. This demonstrates the important proof of principle that enzyme replacement therapy administered directly to the brain ventricles can be effective and presents a desperately awaited ray of hope for CLN2 patients worldwide.”
Additional analysis of cerliponase alfa to treat CLN2 disease will be presented at the International Child Neurology Congress in Amsterdam in May.
Anjali Shukla
