Questions & Answers
On 26 January, the Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion, recommending the granting of a marketing authorisation for the medicinal product Yargesa, intended for treatment of adult patients with mild to moderate type 1 Gaucher disease. The applicant for this medicinal product is JensonR+ Limited.
Yargesa will be available as capsules (100 mg). The active substance of Yargesa 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.
Yargesa is a generic of Zavesca, which has been authorised in the EU since 20 November 2002. Studies have demonstrated the satisfactory quality of Yargesa, and its bioequivalence to the reference product Zavesca.
The full indication is:
“Yargesa is indicated for the oral treatment of adult patients with mild to moderate type 1 Gaucher disease. Yargesa may be used only in the treatment of patients for whom enzyme replacement therapy is unsuitable”.
It is proposed that Yargesa 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 Yargesa||(English only)||2017-01-27|
|INN or common name|
|Therapeutic area||Gaucher Disease|
|Date opinion adopted||26/01/2017|
|Application type||Initial authorisation|
More information on Yargesa
- Questions and answers on generic medicines (2012-12-07)