EU approves ultra-orphan Mepsevii

The European Medicines Agency (EMA) has granted an exceptional Marketing Authorisation (MA) to Mepsevii (vestronidase alfa).

The drug is designed to treat enzyme deficiency disease mucopolysaccharidosis type VII, which has a prevalence of only one in a quarter-of-a-million people.

The EMA only grant around 3 conditional MAs per year, for promising products which require more data collection to properly assess effectiveness. As part of the approval, the company is required to use this opportunity to collect additional data on safety and efficacy.

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Published 30. August 2018 in News, News EU