Posted by Michael Wonder on 02 Jul 2018
Ultragenyx announces positive CHMP opinion for Mepsevii (vestronidase alfa) for the treatment of mucopolysaccharidosis VII
29 June 2018 - Ultragenyx today announced that the CHMP), the scientific committee of the EMA, has adopted a positive opinion recommending the marketing authorisation under exceptional circumstances of Mepsevii (vestronidase alfa) for the treatment of non-neurological manifestations of mucopolysaccharidosis VII (MPS VII; Sly syndrome).
Mepsevii is an enzyme replacement therapy designed to replace the deficient lysosomal enzyme beta-glucuronidase in patients with MPS VII, a progressive and debilitating rare genetic disease.
The CHMP considered that the totality of the data provided for this debilitating orphan disease warranted a recommendation for approval under exceptional circumstances.
