Mepsevii (vestronidase alfa) is a treatment for Mucopolysaccharidosis VII (MPS VII). The Committee for Medicinal Products for Human Use (CHMP) has given a positive recommendation for the marketing authorisation of Mepsevii under exceptional circumstances as a treatment for non-neurological manifestations of MPS VII to the European Commission, which can approve medicines in the European Union. The full article can be found here, at Globe Newswire.
About Mucopolysaccharidosis VII
MPS VII, also known as Sly syndrome, is a lysosomal storage disorder that is caused by the build-up of mucopolysaccharides (a type of carbohydrate) in cells. The condition usually affects most parts of the body, although each individual is different and there is a lot of variation in the symptoms and severity of MPS VII between people. The first signs of the condition usually appear in childhood. Approximately one in every 250,000 children is born with MPS VII, although the precise prevalence is unknown.
Mepsevii is an enzyme replacement therapy designed to treat MPS II. It works by replacing the lysosomal enzyme beta-glucuronidase that is deficient in patients with MPS II. Beta-glucoronidase is involved in the breakdown of certain carbohydrates. Currently, Mepsevii was approved as a treatment for children and adults with MPS II in late 2017.
CHMP’s Positive Recommendation
Ultragenyx Pharmaceutical Inc. has announced that CHMP (a part of the European Medicines Agency) will make a positive recommendation about the marketing authorisation of Mepsevii under exceptional circumstances as a treatment for non-neurological manifestations of MPS VII. This will be reviewed by the European Commission, which can approve medicines in the European Union.
Marketing approval under exceptional circumstances is given when medicines are judged to provide new or better treatment options for patients who have few alternative options. The approval may be granted despite a shortage of information about the drug; for Mepsevii the safety and efficacy data is not comprehensive because MPS II is such a rare condition.
Ultragenyx expects to hear more about the decision of the European Commission in the third quarter of 2018 and is planning to apply to all of the 28 countries in the European Union.