Miglustat Gen.Orph
Authorised
This medicine is authorised for use in the European Union
miglustat
MedicineHumanAuthorised
- Application under evaluation
- CHMP opinion
- European Commission decision
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.
Miglustat Gen.Orph is available as 100 mg capsules to be taken by mouth. The recommended starting dose for type-1 Gaucher disease is one capsule three times a day. For Niemann-Pick type-C disease, it is two capsules three times a day for patients aged 12 years and over; in younger patients, the dose depends on their weight and height. Miglustat Gen.Orph is intended for long-term use.
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 about using Miglustat Gen.Orph, see the package leaflet or contact your doctor or pharmacist.
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.
The active substance in Miglustat Gen.Orph, miglustat, blocks an enzyme called glucosylceramide synthase. 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.
Studies on the benefits and risks of the active substance in Gaucher disease have already been carried out with the reference medicine, Zavesca, and do not need to be repeated for Miglustat Gen.Orph.
As for every medicine, the company provided studies on the quality of Miglustat Gen.Orph. 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.
Because Miglustat Gen.Orph 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.
The European Medicines Agency concluded that, in accordance with EU requirements, Miglustat Gen.Orph has been shown to have comparable quality and to be bioequivalent to Zavesca. Therefore, the Agency’s view was that, as for Zavesca, the benefit of Miglustat Gen.Orph outweighs the identified risk and it can be authorised for use in the EU.
Recommendations and precautions to be followed by healthcare professionals and patients for the safe and effective use of Miglustat Gen.Orph have been included in the summary of product characteristics and the package leaflet.
As for all medicines, data on the use of Miglustat Gen.Orph are continuously monitored. Side effects reported with Miglustat Gen.Orph are carefully evaluated and any necessary action taken to protect patients.
Miglustat Gen.Orph received a marketing authorisation valid throughout the EU on 10 November 2017.
Product information
Latest procedure affecting product information:
N/0000240915
14/01/2025
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).
Product details
- Name of medicine
- Miglustat Gen.Orph
- 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 productsTherapeutic 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.
News on Miglustat Gen.Orph
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