CHMP recommends approval of Mepsevii for mucopolysaccharidosis type VII.- Ultragenyx.
The Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion, recommending the granting of a marketing authorisation for the medicinal product Mepsevii (vestronidase alfa), from Ultragenyx, intended for the treatment of mucopolysaccharidosis type VII. Mepsevii was designated as an orphan medicinal product on 21 March 2012.
Mepsevii will be available as 2 mg/ml concentrate for solution for infusion. The active substance of Mepsevii is vestronidase alfa, a recombinant form of human beta-glucuronidase (ATC code: A16AB18). Mepsevii is an enzyme replacement therapy intended to provide or supplement beta-glucuronidase, an enzyme that helps with the degradation of glycosaminoglycans and thus prevents their accumulation in various tissues in the body. The benefits with Mepsevii are its ability to reduce glycosaminoglycan levels in the body.
The most common side effects are anaphylactoid reaction, urticaria and infusion site swelling. The full indication is: "Mepsevii is indicated for the treatment of non-neurological manifestations of Mucopolysaccharidosis VII (MPS VII; Sly syndrome).
Comment: The FDA approves Mepsevii for Sly Syndrome in November 2017.
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