Mepsevii

vestronidase alfa

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An overview of Mepsevii and why it is authorised in the EU

Mepsevii is a medicine to treat mucopolysaccharidosis type VII (MPS VII, also known as Sly syndrome), an inherited disease caused by a lack of an enzyme needed to break down complex carbohydrates known as glycosaminoglycans (GAGs).

The disease leads to build up of GAGs in the body, which causes a wide range of problems, including joint stiffness, short stature, enlarged liver and spleen, hearing loss, cataract and delays in development.

MPS VII is rare, and Mepsevii was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 21 March 2012. 

How is Mepsevii used?

Mepsevii is given every two weeks as an infusion (drip) into a vein lasting 4 hours. The recommended dose for each infusion is 4 mg per kilogram of body weight. Before the infusion the patient is given antihistamine treatment to reduce the risk of allergic reactions.

The medicine can only be obtained with a prescription and should be given by an appropriately trained healthcare professional who can manage medical emergencies. Treatment should also be supervised by a healthcare professional with experience in the treatment of MPS VII.

For more information about using Mepsevii, see the package leaflet or contact your doctor or pharmacist.

How does Mepsevii work?

Mepsevii is an enzyme replacement therapy that works by replacing the missing enzyme (beta-glucuronidase) in patients with MPS VII, helping to break down GAGs and stopping them building up in the body.

What benefits of Mepsevii have been shown in studies?

Mepsevii has been shown to reduce the levels of GAGs in the body and reduce or stabilise symptoms in patients with MPS VII.

In a main study of 12 patients with MPS VII, patients treated with Mepsevii for 6 months had a 65% reduction in their urine levels of GAGs. In 11 out of 12 patients, symptoms, including those affecting vision and movement, improved or did not get worse.

What are the risks associated with Mepsevii?

The most common side effects with Mepsevii (which may affect more than 1 in 10 people) are anaphylactic reaction (sudden, severe allergic reaction), urticaria (itchy rash) and swelling at the infusion site.

Mepsevii must not be used in patients who have ever had anaphylactic reaction to vestronidase alfa or any of the other ingredients of the medicine. For the full list of side effects and restrictions, see the package leaflet.

Why is Mepsevii authorised in the EU?

Mepsevii reduces the levels of GAGs in the body and improves or at least stabilises the symptoms of MPS VII. In addition, most adverse reactions are mild to moderate in severity.

Although only a limited amount of data is available from clinical studies, the European Medicines Agency took into account the life-threatening and debilitating nature of MPS VII and the lack of authorised medicines. EMA decided that Mepsevii’s benefits are greater than its risks and that it can be authorised for use in the EU.

Mepsevii has been authorised under ‘exceptional circumstances’. This is because it has not been possible to obtain complete information about Mepsevii due to the rarity of the disease and for scientific reasons. Every year, the Agency will review any new information that becomes available and this overview will be updated as necessary.

What information is still awaited for Mepsevii?

Since Mepsevii has been authorised under exceptional circumstances, the company that markets Mepsevii will conduct a study to provide data on the long-term effectiveness and safety of Mepsevii as well as on the disease itself, including its progression and development of symptoms.

What measures are being taken to ensure the safe and effective use of Mepsevii?

Recommendations and precautions to be followed by healthcare professionals and patients for the safe and effective use of Mepsevii have been included in the summary of product characteristics and the package leaflet.

As for all medicines, data on the use of Mepsevii are continuously monitored. Side effects reported with Mepsevii are carefully evaluated and any necessary action taken to protect patients.

Other information about Mepsevii

Mepsevii received a marketing authorisation under exceptional circumstances valid throughout the EU on 23 August 2018.

Name Language First published Last updated
Mepsevii : EPAR - Medicine overview BG = bălgarski 2018-08-28  
Mepsevii : EPAR - Medicine overview ES = español 2018-08-28  
Mepsevii : EPAR - Medicine overview CS = čeština 2018-08-28  
Mepsevii : EPAR - Medicine overview DA = dansk 2018-08-28  
Mepsevii : EPAR - Medicine overview DE = Deutsch 2018-08-28  
Mepsevii : EPAR - Medicine overview ET = eesti keel 2018-08-28  
Mepsevii : EPAR - Medicine overview EL = elliniká 2018-08-28  
Mepsevii : EPAR - Medicine overview EN = English 2018-08-28  
Mepsevii : EPAR - Medicine overview FR = français 2018-08-28  
Mepsevii : EPAR - Medicine overview IT = italiano 2018-08-28  
Mepsevii : EPAR - Medicine overview LV = latviešu valoda 2018-08-28  
Mepsevii : EPAR - Medicine overview LT = lietuvių kalba 2018-08-28  
Mepsevii : EPAR - Medicine overview HU = magyar 2018-08-28  
Mepsevii : EPAR - Medicine overview MT = Malti 2018-08-28  
Mepsevii : EPAR - Medicine overview NL = Nederlands 2018-08-28  
Mepsevii : EPAR - Medicine overview PL = polski 2018-08-28  
Mepsevii : EPAR - Medicine overview PT = português 2018-08-28  
Mepsevii : EPAR - Medicine overview RO = română 2018-08-28  
Mepsevii : EPAR - Medicine overview SK = slovenčina 2018-08-28  
Mepsevii : EPAR - Medicine overview SL = slovenščina 2018-08-28  
Mepsevii : EPAR - Medicine overview FI = suomi 2018-08-28  
Mepsevii : EPAR - Medicine overview SV = svenska 2018-08-28  
Mepsevii : EPAR - Medicine overview HR = Hrvatski 2018-08-28  
Name Language First published Last updated
Mepsevii : EPAR - Risk-management-plan summary (English only) 2018-08-28  

This EPAR was last updated on 28/08/2018 .

Authorisation details

Product details

Product details for Mepsevii
NameMepsevii
Agency product numberEMEA/H/C/004438
Active substance

vestronidase alfa

International non-proprietary name (INN) or common name

vestronidase alfa

Therapeutic area Mucopolysaccharidosis VII
Anatomical therapeutic chemical (ATC) code A16AB18
Additional monitoring

This medicine is under additional monitoring. This means that it is being monitored even more intensively than other medicines. For more information, see medicines under additional monitoring.

Treatment of rare diseases

This medicine has an "orphan designation" which means that it is used to treat life-threatening or chronically debilitating conditions that affect no more than five in 10,000 people in the European Union, or are medicines which, for economic reasons, would be unlikely to be developed without incentives.

Exceptional Circumstances

There were "exceptional circumstances" concerning the approval of this medicine. This happens when the applicant can show that they are unable to provide comprehensive data on the efficacy and safety of the medicine for which authorisation is being sought, due to the rarity of the condition it is intended for, limited scientific knowledge in the area concerned, or ethical considerations involved in the collection of such data.

Publication details

Publication details for Mepsevii
Marketing-authorisation holder

Ultragenyx Germany GmbH

Revision0
Date of issue of marketing authorisation valid throughout the European Union23/08/2018

Contact address:

Ultragenyx Germany GmbH
Stadtquartier Friedrichstrasse 191
10117 Berlin
Germany

Product information

Product information

23/08/2018  Mepsevii -EMEA/H/C/004438

Name Language First published Last updated
Mepsevii : EPAR - Product Information EN = English 2018-08-28  
Mepsevii : EPAR - Product Information EN = English 2018-08-28  
Mepsevii : EPAR - Product Information EN = English 2018-08-28  
Mepsevii : EPAR - Product Information EN = English 2018-08-28  
Mepsevii : EPAR - Product Information EN = English 2018-08-28  
Mepsevii : EPAR - Product Information EN = English 2018-08-28  
Mepsevii : EPAR - Product Information EN = English 2018-08-28  
Mepsevii : EPAR - Product Information EN = English 2018-08-28  
Mepsevii : EPAR - Product Information EN = English 2018-08-28  
Mepsevii : EPAR - Product Information EN = English 2018-08-28  
Mepsevii : EPAR - Product Information EN = English 2018-08-28  
Mepsevii : EPAR - Product Information EN = English 2018-08-28  
Mepsevii : EPAR - Product Information EN = English 2018-08-28  
Mepsevii : EPAR - Product Information EN = English 2018-08-28  
Mepsevii : EPAR - Product Information EN = English 2018-08-28  
Mepsevii : EPAR - Product Information EN = English 2018-08-28  
Mepsevii : EPAR - Product Information EN = English 2018-08-28  
Mepsevii : EPAR - Product Information EN = English 2018-08-28  
Mepsevii : EPAR - Product Information EN = English 2018-08-28  
Mepsevii : EPAR - Product Information EN = English 2018-08-28  
Mepsevii : EPAR - Product Information EN = English 2018-08-28  
Mepsevii : EPAR - Product Information EN = English 2018-08-28  
Mepsevii : EPAR - Product Information EN = English 2018-08-28  
Mepsevii : EPAR - Product Information EN = English 2018-08-28  
Mepsevii : EPAR - Product Information EN = English 2018-08-28  

Contents

  • Annex I - Summary of product characteristics
  • Annex IIA - Manufacturing-authorisation holder responsible for batch release
  • Annex IIB - Conditions of the marketing authorisation
  • Annex IIIA - Labelling
  • Annex IIIB - Package leaflet

Please note that the size of the above document can exceed 50 pages.

You are therefore advised to be selective about which sections or pages you wish to print.


Name Language First published Last updated
Mepsevii : EPAR - All Authorised presentations EN = English 2018-08-28  
Mepsevii : EPAR - All Authorised presentations EN = English 2018-08-28  
Mepsevii : EPAR - All Authorised presentations EN = English 2018-08-28  
Mepsevii : EPAR - All Authorised presentations EN = English 2018-08-28  
Mepsevii : EPAR - All Authorised presentations EN = English 2018-08-28  
Mepsevii : EPAR - All Authorised presentations EN = English 2018-08-28  
Mepsevii : EPAR - All Authorised presentations EN = English 2018-08-28  
Mepsevii : EPAR - All Authorised presentations EN = English 2018-08-28  
Mepsevii : EPAR - All Authorised presentations EN = English 2018-08-28  
Mepsevii : EPAR - All Authorised presentations EN = English 2018-08-28  
Mepsevii : EPAR - All Authorised presentations EN = English 2018-08-28  
Mepsevii : EPAR - All Authorised presentations EN = English 2018-08-28  
Mepsevii : EPAR - All Authorised presentations EN = English 2018-08-28  
Mepsevii : EPAR - All Authorised presentations EN = English 2018-08-28  
Mepsevii : EPAR - All Authorised presentations EN = English 2018-08-28  
Mepsevii : EPAR - All Authorised presentations EN = English 2018-08-28  
Mepsevii : EPAR - All Authorised presentations EN = English 2018-08-28  
Mepsevii : EPAR - All Authorised presentations EN = English 2018-08-28  
Mepsevii : EPAR - All Authorised presentations EN = English 2018-08-28  
Mepsevii : EPAR - All Authorised presentations EN = English 2018-08-28  
Mepsevii : EPAR - All Authorised presentations EN = English 2018-08-28  
Mepsevii : EPAR - All Authorised presentations EN = English 2018-08-28  
Mepsevii : EPAR - All Authorised presentations EN = English 2018-08-28  
Mepsevii : EPAR - All Authorised presentations EN = English 2018-08-28  
Mepsevii : EPAR - All Authorised presentations EN = English 2018-08-28  

Pharmacotherapeutic group

Enzymes

Therapeutic indication

Mepsevii is indicated for the treatment of non-neurological manifestations of Mucopolysaccharidosis VII (MPS VII; Sly syndrome).

Assessment History

Changes since initial authorisation of medicine

Name Language First published Last updated

Initial marketing-authorisation documents

Name Language First published Last updated
Mepsevii : Orphan maintenance assessment report EN = English 2018-08-28  
Mepsevii : EPAR - Public assessment report EN = English 2018-08-28  
CHMP summary of positive opinion for Mepsevii EN = English 2018-06-29  

Authorised

This medicine is approved for use in the European Union

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