Date: 2018-10-18

Type of information: Granting of a Market Authorisation in the EU

Product name: Mepsevii®

Compound: vestronidase alfa-vjbk

Therapeutic area: Rare diseases - Genetic diseases

Action mechanism:

  • enzyme replacement therapy. Mucopolysaccharidosis 7 is caused by a deficiency of the lysosomal enzyme beta-glucuronidase, which is required for the breakdown of certain complex carbohydrates known as glycosaminoglycans (GAGs). The inability to break down GAGs leads to their accumulation in many tissues, resulting in serious disease. Patients with MPS 7 may have abnormal coarsened facial features, enlargement of the liver and spleen, airway obstruction, lung disease, cardiovascular complications, joint stiffness, short stature, and skeletal disease.
  • This lysosomal storage disorder is an extremely rare, inherited, genetic condition and impacts less than 150 patients worldwide. The life expectancy of individuals with MPS VII depends on the severity of symptoms. Some affected individuals do not survive infancy, while others may live into adolescence or adulthood. Heart disease and airway obstruction are major causes of death in people with MPS VII. Affected individuals may have developmental delay and progressive intellectual disability.
  • Glucuronidase (GUS) is an enzyme found in the lysosome that when taken up into cells efficiently by a particular receptor results in the delivery of the GUS enzyme into the lysosomes and clearance of stored GAGs. Preclinical studies of the chronic administration of rhGUS results in a reduction of tissue pathology in the liver, spleen, lung, heart, kidney, muscle, bone and brain.

Company: Ultragenyx Pharmaceutical (USA - CA)

Disease: mucopolysaccharidosis 7 (MPS 7, Sly syndrome)

Latest news:

  • • On October 18, 2018, Ultragenyx Pharmaceutical announced that Brazil’s National Health Surveillance Agency (ANVISA) has approved Mepsevii™ (vestronidase alfa) for the treatment of mucopolysaccharidosis VII (MPS VII; Sly syndrome) for patients of all ages.
  • • On August 27, 2018, the European Commission (EC) has approved the Marketing Authorization Application (MAA) for Mepsevii™ (vestronidase alfa), for the treatment of non-neurological manifestations of Mucopolysaccharidosis VII. Mepsevii™ is now approved for use in all 28 EU countries and in Iceland, Liechtenstein and Norway. The approval follows a positive opinion adopted on June 28, 2018 by the European Committee for Medicinal Products for Human Use (CHMP) to recommend approval of Mepsevii™ under exceptional circumstances.
  • • On November 15, 2017, The FDA approved Mepsevii® (vestronidase alfa-vjbk) to treat pediatric and adult patients with mucopolysaccharidosis type VII (MPS VII), also known as Sly syndrome. The safety and efficacy of Mepsevii® were established in clinical trial and expanded access protocols enrolling a total of 23 patients ranging from 5 months to 25 years of age. Patients received treatment with Mepsevii® at doses up to 4 mg/kg once every two weeks for up to 164 weeks. Efficacy was primarily assessed via the six-minute walk test in ten patients who could perform the test. After 24 weeks of treatment, the mean difference in distance walked relative to placebo was 18 meters. Additional follow-up for up to 120 weeks suggested continued improvement in three patients and stabilization in the others. Two patients in the Mepsevii® development program experienced marked improvement in pulmonary function. Overall, the results observed would not have been anticipated in the absence of treatment. The effect of Mepsevii® on the central nervous system manifestations of MPS VII has not been determined.
  • The most common side effects after treatment with Mepsevii® include infusion site reactions, diarrhea, rash and anaphylaxis.
  • The FDA granted this application Fast Track designation. Mepsevii® also received Orphan Drug designation, which provides incentives to assist and encourage the development of drugs for rare diseases. Ultragenyx Pharmaceutical is receiving a Rare Pediatric Disease Priority Review Voucher under a program intended to encourage development of new drugs and biologics for the prevention and treatment of rare pediatric diseases. This is the twelfth rare pediatric disease priority review voucher issued by the FDA since the program began.
  • The FDA is requiring the manufacturer to conduct a post-marketing study to evaluate the long-term safety of the product.
  • Mepsevii® will be available to patients in the U.S. later this month. In order to support patients, Ultragenyx has launched UltraCare™, a comprehensive support service that will provide ongoing support to patients and caregivers. UltraCare will help patients obtain coverage and assist with financial support for both medication and administration of medication.
  • In Europe, the European Medicines Agency (EMA) is currently reviewing the Marketing Authorization Application (MAA) for vestronidase alfa, and an opinion from the Committee for Medicinal Products for Human Use (CHMP) is expected in the first half of 2018.


Submission of marketing authorization application USA :

Submission of marketing authorization application UE:

Withdrawal of marketing authorization application USA:

Withdrawal of marketing authorization application UE:

US authorization: 2017-11-15

UE authorization: 2018-08-27

Favourable opinion UE: 2018-06-28

Favourable opinion USA:

Orphan status USA: 2012-02-16

Orphan status UE: 2012-03-21

Pediatric exclusivit _USA:

Pediatric exclusivity UE:

OTC status:

Other news:

Is general: Yes