Basic Information
A16AX06
miglustat
Other alimentary tract and metabolism products
Therapeutic indication
Miglustat Dipharma is indicated for the oral treatment of adult patients with mild to moderate type 1 Gaucher disease.
Miglustat Dipharma may be used only in the treatment of patients for whom enzyme replacement therapy is unsuitable.
Miglustat Dipharma is indicated for the treatment of progressive neurological manifestations in adult patients and paediatric patients with Niemann-Pick type C disease.
Overview Summary
Miglustat Dipharma 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 Dipharma 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 Dipharma 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 Dipharma 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
Miglustat Dipharma is a ‘generic medicine’. This means that Miglustat Dipharma contains the same active substance and works in the same way as a ‘reference medicine’ already authorised in the EU called Zavesca.
Active Substances (2)
miglustat
miglustat
Documents (9)
Miglustat Dipharma : EPAR - Procedural steps taken and scientific information after authorisation
June 25, 2025
CHANGES_SINCE_INITIAL_AUTHORISATION
Miglustat Dipharma : EPAR - Procedural steps taken and scientific information after authorisation (archive)
October 14, 2019
CHANGES_SINCE_INITIAL_AUTHORISATION
CHMP summary of positive opinion for Miglustat Dipharma
December 14, 2018
INITIAL_MARKETING_AUTHORISATION_DOCUMENTS
Miglustat Dipharma : EPAR - Public assessment report
May 2, 2019
INITIAL_MARKETING_AUTHORISATION_DOCUMENTS
Miglustat Dipharma : EPAR - Public assessment report
May 1, 2019
CHANGES_SINCE_INITIAL_AUTHORISATION
CHMP summary of positive opinion for Miglustat Dipharma
December 14, 2018
CHANGES_SINCE_INITIAL_AUTHORISATION
Miglustat Dipharma : EPAR - Product information
May 1, 2019
DRUG_PRODUCT_INFORMATION
Miglustat Dipharma : EPAR - Medicine overview
May 1, 2019
OVERVIEW_DOCUMENT
Miglustat Dipharma : EPAR - All authorised presentations
May 1, 2019
AUTHORISED_PRESENTATIONS
Overview Q&A (7)
Question
What are the benefits and risks of Miglustat Dipharma?
Answer
Because Miglustat Dipharma 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.
Question
How is Miglustat Dipharma used?
Answer
Miglustat Dipharma 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 Dipharma 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 Dipharma, see the package leaflet or contact your doctor or pharmacist.
Question
How does Miglustat Dipharma work?
Answer
The active substance in Miglustat Dipharma, 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.
Question
Why is Miglustat Dipharma authorised in the EU?
Answer
The European Medicines Agency concluded that, in accordance with EU requirements, Miglustat Dipharma 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 Dipharma outweighs the identified risk and it can be authorised for use in the EU.
Question
What measures are being taken to ensure the safe and effective use of Miglustat Dipharma?
Answer
Recommendations and precautions to be followed by healthcare professionals and patients for the safe and effective use of Miglustat Dipharma have been included in the summary of product characteristics and the package leaflet.
As for all medicines, data on the use of Miglustat Dipharma are continuously monitored. Side effects reported with Miglustat Dipharma are carefully evaluated and any necessary action taken to protect patients.
Question
Other information about Miglustat Dipharma
Answer
Miglustat Dipharma received a marketing authorisation valid throughout the EU on 18 February 2019.
Question
How has Miglustat Dipharma been studied?
Answer
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 Dipharma.
As for every medicine, the company provided studies on the quality of Miglustat Dipharma. 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.