Ultragenyx Announces Positive CHMP Opinion for Mepsevii™ (vestronidase alfa) For the Treatment of Mucopolysaccharidosis VII

NOVATO, CA, USA I June 29, 2018 I Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) today announced that the Committee for Medicinal Products for Human Use (CHMP), the scientific committee of the European Medicines Agency (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.

“This positive CHMP opinion takes us closer to bringing Mepsevii to patients across Europe who are living with this ultra-rare, highly debilitating disease and currently have no approved treatment options,” said Emil D. Kakkis, M.D., Ph.D., Chief Executive Officer and President of Ultragenyx. “This important regulatory milestone underscores our commitment to developing innovative medicines for patients around the world with both rare and even ultra-rare diseases for which the investment and development of the science into first-ever medicines has not yet happened.”   

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.


MPS VII is a progressive rare genetic, metabolic lysosomal storage disorder (LSD) caused by the deficiency of beta-glucuronidase, an enzyme required for the breakdown of the glycosaminoglycans (GAGs) dermatan sulfate, chondroitin sulfate and heparan sulfate. These complex GAG carbohydrates are a critical component of many tissues. The inability to properly break down GAGs leads to a progressive accumulation in many tissues and results in multi-system tissue and organ damage. MPS VII symptoms can include an abnormally coarsened face, pulmonary disease, cardiovascular complications, hepatosplenomegaly (in which the liver and spleen swell beyond their normal size), joint stiffness, short stature, cognitive impairment and the skeletal disease known as dysostosis multiplex.

MPS VII is one of the rarest MPS disorders, affecting an estimated 200 patients in the developed world.

About Mepsevii™ (vestronidase alfa)


Mepsevii is indicated in pediatric and adult patients for the treatment of mucopolysaccharidosis VII (MPS VII, Sly syndrome).

About Ultragenyx

Ultragenyx is a biopharmaceutical company committed to bringing to patients novel products for the treatment of rare and ultra-rare diseases, with a focus on serious, debilitating genetic diseases. Founded in 2010, the company has rapidly built a diverse portfolio of approved therapies and product candidates aimed at addressing diseases with high unmet medical need and clear biology for treatment, for which there are no approved therapies.

The company is led by a management team experienced in the development and commercialization of rare disease therapeutics. Ultragenyx’s strategy is predicated upon time and cost-efficient drug development, with the goal of delivering safe and effective therapies to patients with the utmost urgency.

For more information on Ultragenyx, please visit the company’s website at www.ultragenyx.com.

SOURCE: Ultragenyx Pharmaceutical

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