EU approves Vimizim for MPS IVA - BioMarin

BioMarin Pharmaceutical has announced the European Commission has granted marketing authorization for Vimizim (elosulfase alfa), the first specific treatment approved in the European Union for Mucopolysaccharidosis type IVA (MPS IVA; Morquio A syndrome) in patients of all ages. As the first drug ever approved for Morquio A syndrome, Vimizim has been granted orphan drug status in the European Union, which confers ten years of market exclusivity.

The safety and efficacy of Vimizim were assessed in a 24-week, randomized, double-blind, placebo-controlled clinical trial of 176 patients with MPS IVA ages 5 to 57 years old. The primary endpoint of the trial, change in six-minute walk distance at 24 weeks, was statistically significant in patients receiving weekly infusions at the dose of 2 mg/kg with a mean increase of 22.5 meters over placebo.

The FDA approved Vimizim for patients with Mucopolysaccharidosis type IVA (MPS IVA; Morquio A syndrome) on February 14, 2014. BioMarin has also submitted marketing applications for Vimizim in Brazil, Australia, Canada, Mexico, and Japan.