European Commission approves Mepseviito treat non-neurological manifestations of Mucopolysaccharidosis VII (MPS VII; Sly syndrome).- Ultragenyx Pharma.

Ultragenyx Pharmaceutical Inc. announced that the European Commission (EC) has approved the Marketing Authorization Application (MAA) for Mepsevii (vestronidase alfa), for the treatment of non-neurological manifestations of Mucopolysaccharidosis VII (MPS VII; Sly syndrome).

Mepsevii is now approved for use in all 28 EU countries and in Iceland, Liechtenstein and Norway. The approval follows a positive opinion adopted on June 28, 2018 by the European Committee for Medicinal Products for Human Use (CHMP) to recommend approval of Mepsevii under exceptional circumstances. The European Medicines Agency (EMA) granted Orphan Drug designation to Mepsevii in March 2012. Mepsevii was approved by the FDA for the treatment of pediatric and adult patients with MPS VII in November 2017.

MPS VII (Sly syndrome) is a progressive, rare, genetic, metabolic lysosomal storage disorder (LSD) caused by the deficiency of beta-glucuronidase, an enzyme required for the breakdown of the glycosaminoglycans (GAGs) dermatan sulphate, chondroitin sulphate and heparan sulphate. These complex GAG carbohydrates are a critical component of many tissues. The inability to properly break down GAGs leads to a progressive accumulation in many tissues and results in multisystem tissue and organ damage. MPS VII symptoms can include an abnormally coarsened face, pulmonary disease, cardiovascular complications, hepatosplenomegaly (in which the liver and spleen swell beyond their normal size), joint stiffness, short stature, cognitive impairment and the skeletal disease known as dysostosis multiplex. MPS VII is one of the rarest MPS disorders, affecting an estimated 200 patients in the developed world.