Ultragenyx announces aproval of Mepsevii in Europe for Sly syndrome

Ultragenyx Pharmaceutical 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. Mepsevii is now approved for use in all 28 EU countries and in Iceland, Liechtenstein and Norway.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 D. Kakkis, M.D., Ph.D., Chief Executive Officer and President of Ultragenyx. “This would not have been possible without the commitment and dedication of the patients, their families, and the physicians who participated in our clinical program, and I would like to thank them.”The approval follows a positive opinion adopted on June 28 by the European Committee for Medicinal Products for Human Use,CHMP, to recommend approval of Mepsevii under exceptional circumstances. The European Medicines Agency 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.

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