Ultragenyx Announces Positive CHMP Opinion For Mepsevii

(RTTNews) - Ultragenyx Pharmaceutical Inc. (RARE) said that the Committee for Medicinal Products for Human Use or CHMP, the scientific committee of the European Medicines Agency or EMA, has adopted a positive opinion recommending the marketing authorization 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's positive opinion will now be reviewed by the European Commission (EC), which has the authority to approve medicines for the European Union (EU). The EC decision is expected in the third quarter of 2018 and will apply to all 28 countries of the European Union, Switzerland, Norway, Iceland and Liechtenstein.

The CHMP considered that the totality of the data provided for this debilitating orphan disease warranted a recommendation for approval under exceptional circumstances. This type of authorization can be granted for medicines that offer new or improved treatment options for patients with no or only limited alternatives, in cases where the company is not able to provide comprehensive data.

In the case of Mepsevii, data on the efficacy and safety are limited due to the extreme rarity of the disease, and the marketing authorization requires collection and submission of further data on the clinical efficacy and safety, which will be reviewed by the Committee.

Mepsevii was approved by the U.S. Food and Drug Administration for the treatment of children and adults with MPS VII in November 2017.