Rare Daily Staff

The European Commission has approved Ultragenyx Pharmaceutical’s Mepsevii, the first treatment for children and adults with Mucopolysaccharidosis VII, an ultra-rare metabolic disease that causes progressive damage to organs throughout the body.

The approval of Mepsevii is for the non-neurological manifestations of Mucopolysaccharidosis VII, also known as Sly syndrome. Mepsevii is now approved for use in all 28 EU countries and in Iceland, Liechtenstein, and Norway.

MPS VII is one of the rarest MPS disorders, with an estimated 200 patients in the developed world. It is a lysosomal storage disease that results from the inability of the body to produce adequate amounts of an enzyme needed to rid the body of waste products from the breakdown of carbohydrates. As improperly broken-down fragments of waste accumulate in cells through the body, they cause a breakdown in function.

Mepsevii is an enzyme replacement therapy designed to replace the deficient enzyme beta-glucuronidase in MPS VII patients.

“Dr. William Sly’s science for this treatment has been around for over 25 years and we are honored to be able to develop and finally make this medicine available to MPS VII patients and families in Europe, people who did not know whether a therapy would ever be available to them,” said Emil Kakkis, CEO and president of Ultragenyx.

The approval follows a positive opinion adopted on June 28, 2018 by the European Committee for Medicinal Products for Human Use 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 U.S. Food and Drug Administration for the treatment of pediatric and adult patients with MPS VII in November 2017.

August 29, 2018