CHMP recommends Vimizim for Morquio A Syndrome - BioMarin

The Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion, recommending the granting of a marketing authorisation for the medicinal product Vimizim, 1 mg/ml concentrate for solution for infusion from BioMarin and intended for treatment of mucopolysaccharidosis, type IVA (Morquio A Syndrome, MPS IVA). Elosulfase alfa is intended to supplement the deficit enzyme N-acetylgalactosamine-6-sulfatase. After intravenous administration the enzyme will be taken up into the lysosomes and will increase the catabolism of the glycosaminoglycans (GAGs). The benefits with Vimizim are its ability to improve patient�s 6 minute walking test, and to improve other symptoms of Morquio disease, such as respiratory or anthropometric functions. The most common side effects are infusion reactions, including anaphylaxis, hypersensitivity and vomiting. A pharmacovigilance plan for Vimizim will be implemented as part of the marketing authorisation.

Vimizim was FDA approved on 14 February 2014.