Ultragenyx’ Mepsevii Receives European Approval for MPS 7
Ultragenyx’ Mepsevii (vestronidase alfa) was approved in Europe for the treatment of mucopolysaccharidosis VII (MPS 7), also known as Sly syndrome.
The approval comes after the EMA’s Committee for Medicinal Products for Human Use adopted a positive opinion of the treatment on June 28.
The EMA previously granted Mepsevii orphan drug designation in 2012, and it was approved by the U.S. in November 2017.
Sly syndrome is a rare genetic storage disorder caused by an enzyme deficiency, affecting only an estimated 200 patients worldwide.