BioMarin: European Commission Okays Vimizim To Treat Morquio A Syndrome

(RTTNews) - BioMarin Pharmaceutical Inc (BMRN) said Monday the European Commission has granted marketing authorization for Vimizim, 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 (elosulfase alfa) has been granted orphan drug status in the European Union, which confers ten years of market exclusivity.

"This approval of Vimizim in Europe is a key milestone because we estimate that 85% of Morquio A or MPS IVA patients live outside of the United States," said Jean-Jacques Bienaime, CEO of BioMarin.

The U.S. Food and Drug Administration approved Vimizim for patients with Morquio A Syndrome on February 14, 2014. BioMarin has also submitted marketing applications for Vimizim in Brazil, Australia, Canada, Mexico, and Japan.

Vimizim is an enzyme replacement therapy for the treatment of patients with the lysosomal storage disorder Morquio A syndrome, which is an ultra-rare, severely debilitating disease that affects an estimated 3,000 patients in the developed world.

The disease occurs as a result of a deficiency of activity in an enzyme involved in glycosaminoglycan (GAG) metabolism. The pervasive and progressive accumulation of GAGs leads to significant morbidities and multisystemic clinical impairments resulting in diminished functional capacity, impaired quality of life, and early mortality.