Yargesa

Authorised

This medicine is authorised for use in the European Union

miglustat

Overview

This is a summary of the European public assessment report (EPAR) for Yargesa. It explains how the Agency assessed the medicine to recommend its authorisation in the EU and its conditions of use. It is not intended to provide practical advice on how to use Yargesa.

For practical information about using Yargesa, patients should read the package leaflet or contact their doctor or pharmacist.

What is Yargesa and what is it used for?

Yargesa is a medicine used to treat adults with mild to moderate type-1 Gaucher disease.

Patients with this disease lack an enzyme that breaks down a type of fat called glucosylceramide. As a result, glucosylceramide builds up in different parts of the body, such as the spleen, liver and bones. Yargesa is used in patients who cannot receive enzyme-replacement therapy.

Yargesa is a ‘generic medicine’. This means that it contains the same active substance (miglustat) and works in the same way as a ‘reference medicine’ already authorised in the European Union (EU) called Zavesca.

How is Yargesa used?

Yargesa is available as 100 mg capsules to be taken by mouth. The recommended starting dose is one capsule three times a day. A lower dose should be used in patients with reduced kidney function and those who develop diarrhoea.

The medicine can only be obtained with a prescription and treatment should be supervised by doctors who are experienced in the management of Gaucher disease.

For more information, see the package leaflet.

How does Yargesa work?

The active substance in Yargesa, miglustat, prevents an enzyme called glucosylceramide synthase from working. This enzyme is involved in the first step of the production of glucosylceramide. By preventing the enzyme from working, miglustat can reduce the production of glucosylceramide in cells, thereby reducing the symptoms of type-1 Gaucher disease.

How has Yargesa been studied?

Studies on the benefits and risks of the active substance in the approved use have already been carried out with the reference medicine, Zavesca, and do not need to be repeated for Yargesa.

As for every medicine, the company provided studies on the quality of Yargesa. The company also carried out a study that showed that it is ‘bioequivalent’ to the reference medicine. Two medicines are bioequivalent when they produce the same levels of the active substance in the body and are therefore expected to have the same effect.

What are the benefits and risks of Yargesa?

Because Yargesa is a generic medicine and is bioequivalent to the reference medicine, its benefits and risks are taken as being the same as the reference medicine’s.

Why is Yargesa approved?

The Agency’s Committee for Medicinal Products for Human Use (CHMP) concluded that, in accordance with EU requirements, Yargesa has been shown to have comparable quality and to be bioequivalent to Zavesca. Therefore, the CHMP’s view was that, as for Zavesca, the benefit outweighs the identified risk. The Committee recommended that Yargesa be approved for use in the EU.

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

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

Other information about Yargesa

The European Commission granted a marketing authorisation valid throughout the European Union for Yargesa on 22 March 2017.

For more information about treatment with Yargesa, read the package leaflet (also part of the EPAR) or contact your doctor or pharmacist.

Yargesa : EPAR - Summary for the public

Yargesa : EPAR - Risk management plan summary

Product information

Yargesa : EPAR - Product Information

Yargesa : EPAR - All Authorised presentations

Product details

• Name of medicine:
  Yargesa
• Active substance:
  miglustat
• International non-proprietary name (INN) or common name:
  miglustat
• Therapeutic area (MeSH):
  Gaucher Disease
• Anatomical therapeutic chemical (ATC) code:
  A16AX06

Pharmacotherapeutic group

Other alimentary tract and metabolism products

Therapeutic indication

Yargesa is indicated for the oral treatment of adult patients with mild to moderate type 1 Gaucher disease.

Yargesa may be used only in the treatment of patients for whom enzyme replacement therapy is unsuitable.

Yargesa is indicated for the treatment of progressive neurological manifestations in adult patients and paediatric patients with Niemann-Pick type C disease.

Authorisation details

• EMA product number:
  EMEA/H/C/004016
• Generic:

  This is a generic medicine, which is developed to be the same as a medicine that has already been authorised, called the reference medicine. A generic medicine contains the same active substance(s) as the reference medicine, and is used at the same dose(s) to treat the same disease(s). For more information, see Generic and hybrid medicines.

• Marketing authorisation holder:
  Piramal Critical Care B.V.

  Rouboslaan 32; 2252 TR Voorschoten; Netehrlands

• Opinion adopted:
  26/01/2017
• Marketing authorisation issued:
  22/03/2017
• Revision:
  5

Assessment history

Changes since initial authorisation of medicine

Yargesa : EPAR - Procedural steps taken and scientific information after authorisation

Initial marketing authorisation documents

Yargesa : EPAR - Public assessment report

Reference Number: EMA/103540/2017

CHMP summary of positive opinion for Yargesa

Reference Number: EMA/CHMP/22018/2017