BioMarin Pharmaceutical Inc. has submitted a Marketing Authorization Application (MAA) to the European Medicines Agency for Aryplase (galsulfase), an investigational enzyme replacement therapy for the treatment of mucopolysaccharidosis VI (MPS VI).
Aryplase is an investigational enzyme replacement therapy for the treatment of MPS VI. Aryplase is designed to address the underlying deficiency of MPS VI disease and provide the enzyme that people with MPS VI are lacking.
BioMarin has received orphan medicinal product designation for Aryplase in the European Union.
Emil Kakkis, senior VP of Business Operations at BioMarin, commented, "This milestone represents an important step in our goal to bring the first specific therapeutic treatment option to MPS VI patients around the world. Our ability to submit this European application just seven days after filing for Aryplase marketing authorization in the United States demonstrates our ability to prepare separate and comprehensive marketing authorization applications simultaneously."
MPS VI, also known as Maroteaux-Lamy Syndrome, is an inherited debilitating, life-threatening disease for which no drug therapies are currently available. The majority of people with MPS VI die from disease-related complications between childhood and early adulthood.