Basic Information
Miglustat Dipharma
Regulatory Information
EMEA/H/C/004904
February 18, 2019
December 13, 2018
7
July 25, 2023
Company Information
Germany
Offheimer Weg 33 65549 Limburg a. d. Lahn
DIPHARMA Arzneimittel GmbH
Drug Classification
Active Substances Detail
EMA Resources
Detailed Information
Therapeutic Indication
### 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.