New Drug Gets Positive Opinion for Approval in EU

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Zavesca (formerly known as Vevesca or OGT 918) has received a positive opinion recommending approval in the European Union for use in patients with mild to moderate Type 1 Gauchers disease with the proviso that it is used by patients for whom enzyme replacement therapy is unsuitable and that follow-up safety data is obtained on all patients.

On 26 July 2002 the Committee for Proprietary Medicinal Products (CPMP) of the European Agency for the Evaluation of Medicinal Products (EMEA) issued a positive opinion recommending approval of Zavesca to patients with mild to moderate disease Type 1 Gauchers disease for whom enzyme replacement therapy is unsuitable.

All patients will have to be monitored regarding potential side effects. Marketing Authorisation will be granted subject to certain obligations including a request that follow-up safety data from a post-marketing surveillance programme and additional clinical information are provided.

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

Trials have shown that the spleen and liver size of patients with Gauchers disease have decreased and haemoglobin and, to a lesser extent, platelet counts have increased. However side affects noted during the trials include diarrhoea, loss of weight and tremor. Cases of peripheral neuropathy (increased or loss of sensation firstly in the hands or feet) have also been reported with or without concurrent conditions such as vitamin B12 deficiency and monoclonal gammopathy (antibodies which are secreted by abnormal cells in the marrow).

The CPMP opinion serves as the basis for a European Commission (EC) final approval which is typically issued after 90 to 120 days. EC approval would result in a single marketing authorisation valid in all 15 EU member states.

To ensure rapid availability of Zavesca to patients in need in the European Union, Oxford GlycoSciences (OGS) has signed a binding letter of intent with another pharmaceutical company, Actelion Ltd, setting out the conditions for a European marketing and distribution partnership. Actelion is experienced in providing drug safety and post-marketing surveillance.

FDA Delays Approval in the US

The Food and Drugs Administration (FDA) has stated that the application to market Zavesca in the US may only be approved if a number of issues are addressed and further clinical studies conducted.

'Zavesca is a major investment for OGS and we plan to expand the world-wide clinical trial programme and continue our clinical trials in the US', says David Ebsworth, OGS's newly appointed Chief Executive Officer. A trial is currently being carried out on Type 1 patients by Dr Gregory Pastores at the NYU Medical Centres in New York. A trial of Type 3 patients is being considered at another centre.

EWGGD Advisory Council

The European Working Group on Gaucher Disease has convened an Advisory Council to develop a Consensus Document on the treatment of Type 1 Gaucher disease specifically to discuss Zavesca as an option. The Council consists of physicians representing many European countries and two representatives from the European Gaucher Alliance.

The Consensus Document will focus specifically on the treatment of Type 1 Gaucher disease but will also cover Type 2 and 3. The Document will be discussed at a meeting to coincide with the Royal Society (London) meeting on Glycolipids in Cell Biology and Medicine to be held on 16-17 October 2002.

It is envisaged that a paper will then be submitted for publication in a peer-reviewed journal. The Advisory Council is being supported by an unrestricted educational grant from Oxford GlycoSciences.

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Source: Gauchers News October 2002.
© Copyright Gauchers Association 2002