BioMarin Submits Vimizim in Canada

BioMarin Pharmaceutical has submitted a New Drug Submission (NDS) for Vimizim (elosulfase alfa) under Priority Review status. Vimizim is an investigational enzyme replacement therapy for the treatment of patients with the lysosomal storage disorder Morquio A syndrome, also called Mucopolysaccharidosis Type IVA (MPS IVA). Approval of Vimizim would make it the first available drug treatment option for Canadians who have this rare genetic metabolic disorder.

Health Canada designates Priority Review to drugs that offer major advances in treatment or provide a treatment where no adequate therapy exists. Priority is intended to accelerate the regulatory review.

Vimizim is currently under review by the FDA for patients with Morquio A syndrome, or MPS IVA. Vimizim is the first and only enzyme replacement therapy (ERT) designed to target the underlying cause of Morquio A Syndrome, a deficiency in the enzyme N-acetylgalactosamine-6 sulfatase (GALNS). Infused enzyme replacement therapy with Vimizim replaces deficient GALNS activity to minimize progressive multi-systemic manifestations.