Miglustat Gen.Orph

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On 14 September 2017, the Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion, recommending the granting of a marketing authorisation for the medicinal product Miglustat Gen.Orph, intended for treatment of adult patients with mild to moderate type 1 Gaucher disease. The applicant for this medicinal product is Gen.Orph.

Miglustat Gen.Orph will be available as capsules (100 mg). The active substance of Miglustat Gen.Orph is miglustat, an inhibitor of glucosylceramide synthase, the enzyme responsible for the first step in the synthesis of glycosphingolipids (ATC code: A16AX06). It works as substrate reduction therapy. This means that it reduces production of glycosphingolipids, the substrates of the defective enzyme in patients with Gaucher disease (glucocerebrosidase). Reducing glycosphingolipids levels is expected to slow down or prevent symptoms of type 1 Gaucher disease.

Miglustat Gen.Orph is a generic of Zavesca which has been authorised in the EU since 20 November 2002. Studies have demonstrated the satisfactory quality of Miglustat Gen.Orph, and its bioequivalence to the reference product Zavesca.

The full indication is:

“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”.

It is proposed that Miglustat Gen.Orph be prescribed by physicians who are knowledgeable in the management of Gaucher disease.

Detailed recommendations for the use of this product will be described in the summary of product characteristics (SmPC), which will be published in the European public assessment report (EPAR) and made available in all official European Union languages after the marketing authorisation has been granted by the European Commission.

Name Language First published Last updated
CHMP summary of positive opinion for Miglustat Gen.Orph (English only) 2017-09-15  

Key facts

Product details for Miglustat Gen.Orph
NameMiglustat Gen.Orph
INN or common namemiglustat
Therapeutic area Gaucher Disease
Active substance

miglustat

Date opinion adopted14/09/2017
Company name

Gen.Orph

StatusPositive
Application typeInitial authorisation

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