Miglustat Gen.Orph
miglustat
Table of contents
Overview
Miglustat Gen.Orph is used to treat two inherited diseases that affect the way the body handles fats. Both diseases cause a build-up of fatty substances called glycosphingolipids in the body. Miglustat Gen.Orph is used to treat the following patients:
- adults (aged 18 years and above) with mild to moderate type-1 Gaucher disease. Patients with this disease lack an enzyme called glucocerebrosidase, which results in a glycosphingolipid called glucosylceramide building up in different parts of the body, such as the spleen, liver and bones. Miglustat Gen.Orph is used in patients who cannot receive the standard treatment of enzyme-replacement therapy (ERT);
- patients of all ages with Niemann-Pick type-C disease, a potentially fatal disease in which glycosphingolipids build up within cells in the brain and elsewhere in the body. Miglustat Gen.Orph is used to treat the neurological symptoms of the disease (symptoms affecting the brain and nerves). These include loss of coordination, problems with ‘saccadic’ (rapid) eye movements that can lead to impaired vision, delayed development, difficulty swallowing, decreased muscle tone, fits and learning difficulties.
Miglustat Gen.Orph is a ‘generic medicine’. This means that Miglustat Gen.Orph contains the same active substance and works in the same way as a ‘reference medicine’ already authorised in the EU called Zavesca.
Authorisation details
| Product details | |
|---|---|
| Name |
Miglustat Gen.Orph
|
| Agency product number |
EMEA/H/C/004366
|
| Active substance |
miglustat
|
| International non-proprietary name (INN) or common name |
miglustat
|
| Therapeutic area (MeSH) |
Gaucher Disease
|
| Anatomical therapeutic chemical (ATC) code |
A16AX06
|
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. |
| Publication details | |
|---|---|
| Marketing-authorisation holder |
Gen.Orph
|
| Revision |
9
|
| Date of issue of marketing authorisation valid throughout the European Union |
09/11/2017
|
| Contact address |
185 Bureaux de la Colline
92213 Saint Cloud Cedex France |
Product information
13/03/2023 Miglustat Gen.Orph - EMEA/H/C/004366 - IB/0024
This medicine’s product information is available in all official EU languages.
Select ‘available languages’ to access the language you need.
Product information documents contain:
- summary of product characteristics (annex I);
- manufacturing authorisation holder responsible for batch release (annex IIA);
- conditions of the marketing authorisation (annex IIB);
- labelling (annex IIIA);
- package leaflet (annex IIIB).
You can find product information documents for centrally authorised human medicines on this website. For centrally authorised veterinary medicines authorised or updated from February 2022, see the Veterinary Medicines Information website.
Pharmacotherapeutic group
Other alimentary tract and metabolism products
Therapeutic indication
Miglustat Gen.Orph is indicated for the oral treatment of adult patients with mild to moderate type 1 Gaucher disease.
Miglustat Gen.Orph may be used only in the treatment of patients for whom enzyme replacement therapy is unsuitable.
Miglustat Gen.Orph is indicated for the treatment of progressive neurological manifestations in adult patients and paediatric patients with Niemann-Pick type C disease.