BioMarin Pharmaceutical Inc. announced that ANVISA, Brazil's National Health Surveillance Agency, has granted BioMarin marketing approval for Naglazyme (galsulfase) for the treatment of patients with Mucopolysaccharidosis VI (MPS VI).
"We are very pleased to receive marketing approval for Naglazyme in Brazil which has the largest known number of affected MPS VI patients in the world," said Stephen Aselage, senior vice president of global commercial development at BioMarin.
"Although there is no formal orphan drug designation in Brazil, ANVISA approved Naglazyme in just eight months based on a recent resolution that gives priority review for therapies that are developed for the treatment of neglected diseases or are considered orphan disorders.
This emphasizes the need to rapidly bring life-altering therapies to patients around the world, and we remain dedicated to pursuing continued geographic expansion and are optimistic regarding the long-term potential of Naglazyme."
Naglazyme was approved by the US Food and Drug Administration (FDA) in May 2005 and by the European Commission (EC) in January 2006. As the first drug approved for MPS VI, the FDA and EC have both designated Naglazyme as an orphan drug, conferring seven years of market exclusivity in the United States and 10 years of market exclusivity in the European Union.
MPS VI (also known as Maroteaux-Lamy syndrome) is a debilitating, life-threatening genetic disease caused by a deficiency of the enzyme N-acetylgalactosamine 4-sulfatase. This enzyme deficiency leads to the accumulation of certain complex carbohydrates, glycosaminoglycans (GAGs), in the lysosomes, giving rise to progressive cellular, tissue and organ system dysfunction. The majority of individuals with MPS VI die from disease-related complications between childhood and early adulthood.
Naglazyme is the first and only enzyme replacement therapy indicated for the treatment of MPS VI. Naglazyme is indicated for patients with MPS VI. Naglazyme has been shown to improve walking and stair-climbing capacity.
BioMarin develops and commercializes innovative biopharmaceuticals for serious diseases and medical conditions. The company's product portfolio comprises three approved products and multiple clinical and preclinical product candidates.