Zavesca receives EU Marketing Authorisation

Gauchers News Contents

Zavesca, a capsule taken by mouth three times a day, received European Commission approval on 26 November 2002 for the treatment of patients with mild to moderate type 1 Gauchers disease for whom enzyme replacement therapy is unsuitable. Patients taking the drug should be managed by physicians knowledgeable about Gauchers disease.

Oxford GlycoSciences, which developed the drug following pioneering work by Dr Fran Platt and Dr Terry Butters at Oxford Glycobiology Institute, University of Oxford, has transferred marketing authorisation to Actelion Ltd, a Swiss bio-pharmaceutical company. Actelion began its launch of the drug in the UK on 3 March 2003.

As is usual for orphan drugs, the European Commission has granted Zavesca marketing authorisation under exceptional circumstances, requesting that follow-up safety data derived from a post-marketing surveillance programme and additional clinical information is provided to the authorities on a regular basis.

In a number of clinical trials, Zavesca was shown to reverse many of the clinical signs and laboratory hallmarks of Gauchers disease.

Zavesca (previously known as OGT 918) is a small molecule drug which acts as an oral inhibitor of a key enzyme called glucosylceramide synthase. The rationale for the use of the drug is to help reduce the overall level of glycophingolipid (fatty material) which accumulates in the spleen, liver, bone marrow and sometimes other places in patients with Gauchers disease.

Surveillance Programme

During recent trials of OGT 918 (Zavesca) in Type 1 Gauchers disease, it was observed that most patients experienced gastrointestinal disorders such as diarrhoea and flatulence, weight loss and dizziness. Most of these resolved after a period of time.

In addition several patients had trembling and a few developed a tingling, burning or numb sensation in their hands and feet. One patient experienced memory loss but investigation indicated that this was unlikely to be related to the drug.

The surveillance programme is aimed to find out more about these latter symptoms. Patients taking the drug will be asked to undergo a series of tests as given below.

In addition a number of adults with Gauchers disease who are not taking Zavesca have been asked to participate in an Observational Study. These are currently taking place in two Gauchers Centres, one in the UK and one in Holland. Another UK Centre is due to participate shortly.

Apart from routine assessments which are undertaken as part of a six month routine follow-up, patients will be asked to take part in:

Patients may be asked to repeat some of the tests at regular intervals.

Advisory Council

The European Working Group on Gauchers Disease (EWGGD) has established an Advisory Council of doctors, scientists and patient representatives which has prepared a Position Paper including the criteria as to who would be suitable candidates for the drug and what tests are necessary. The EWGGD will also oversee the Observational Study.

Gauchers News Contents

Source: Gauchers News April 2003. Updated link April 2004.
© Copyright Gauchers Association 2003.