SILVER SPRING, Md. — The Food and Drug Administration has approved Vimizim (elosulfase alfa), the first FDA-approved treatment for Mucopolysaccharidosis Type IVA (Morquio A syndrome), which is a rare disease caused by a deficiency in N-acetylgalactosamine-6-sulfate sulfatase (GALNS).
Vimizim is intended to replace the missing GALNS enzyme involved in an important metabolic pathway. The absence of the enzyme leads to problems with bone development, growth and mobility. There are approximately 800 patients with Morquio A syndrome in the United States. Vimizim also is the first drug to receive the Rare Pediatric Disease Priority Review Voucher, which seeks to encourage development of new drugs and biologics that prevent and treat rare pediatric diseases.
“This approval and rare pediatric disease priority review voucher underscores the agency’s commitment to making treatments available to patients with rare diseases,” said Andrew E. Mulberg, M.D., deputy director of the Division of Gastroenterology and Inborn Errors Products in the FDA’s Center for Drug Evaluation and Research. “Prior to today’s approval, patients with this rare disease have had no approved drug treatment options.”
Vimizim is marketed by BioMarin Pharmaceutical.