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OGT 918, a substrate balance (deprivation) therapy, which is currently on trial for treatment of Gaucher's disease, has been re-named Vevesca. The drug was discussed at the European Working Group for Gaucher Disease meeting in Jerusalem on 6-9 September 2000 and at the National Gaucher Foundation Conference in Arlington, Virginia on 12-13 November 2000. OGT 918 was designated an Orphan Medicinal Product by the European Commission on 18 October 2000. Susan Lewis reports on the progress so far:
Prof Ari Zimran, Director of the Gaucher Clinic at the Shaare-Zedek Medical Centre, Jerusalem (pictured) said at meetings in Jerusalem and in Arlington that preliminary data on OGT 918 (Vevesca) indicates that it appears to be a promising treatment for Gaucher's disease and other lysosomal diseases.
He said: 'It has always been the hope of patients to take a pill to treat their symptoms. The main problems with taking enzyme replacement therapy have been the need for life long dependency on intravenous infusions and the high cost which makes it difficult for patients to obtain in several countries. Pulmonary hypertension may also be a complication of the treatment for some patients.
'I believe there is a place for OGT 918 in the treatment of Type 1 Gaucher disease. However some parameters may take longer to respond than with enzyme replacement therapy to show comparable results.'
Prof Zimran described the first trial of OGT 918 which is published in the Lancet (full details in Gaucher's News, August 2000). He then presented data on 18 patients who continued to take the drug as part of an extension trial. At 18 months these patients showed further significant reductions in liver and spleen volume together with continued significant improvements in haemoglobin and platelets, compared to baseline (that is when they started treatment).
He explained that the Gaucher Clinic at the Shaare-Zedek Medical Centre is also carrying out a 'switch-over' study in patients who have been treated with enzyme replacement therapy for at least two years where:
One third continue with enzyme replacement therapy (control group)
One third switch to OGT 918 (switch group)
One third have a combination of the two drugs (co-administration group).
On 12 February 2001 Oxford GlycoSciences, the company developing the drug, announced initial results of the switch-over study: 'The initial results indicate that patients were successfully maintained on oral therapy alone during the six month study period.
'Of the 33 patients (out of 36) who completed the trial, 29 elected to enter a Vevesca monotherapy extension protocol, comprising ten from the switch group, ten from the control group and nine from the co-administration group.'
A further trial with 18 patients is taking place in Dr Rene Heitners clinic in Johannesburg, South Africa, and in Prof Zimran's Clinic in Israel. These patients are taking a lower dose of OGT 918 where a 50mg pill is given three times a day instead of a 100mg pill taken three times daily.
Prof Zimran made clear that the effectiveness of the drug and possible side effects cannot be fully evaluated until all the trials are completed, the data analysed and the details published.
'We are encouraged by the preliminary analysis of the switch-over study which shows evidence of the benefits of Vevesca (OGT 918) and of patients' preference for oral maintenance therapy,' he said.
'We have shown that our patients can be switched to Vevesca for at least six months - liberating them from the burden of regular intravenous enzyme infusions.
'He said that it was important that there should be more than a single treatment for Gaucher's disease and that research should continue to improve the medical management of patients.
OGT 918 (Vevesca) was designated as an Orphan Medicinal Product on 18 October 2000 by the European Commission for the treatment of Gaucher's disease.
Oxford GlycoSciences (OGS) is one of the first companies to have a drug designated as Orphan in the European Union (EU) under new legislation that became law earlier this year.
The Committee stated there is no satisfactory treatment for Types 2 and 3 Gaucher's disease and although there is a treatment (enzyme replacement therapy) for Type 1, Vevesca may provide benefit to these patients.
The designation allows for a ten year marketing exclusivity period in the EU following approval of Vevesca.
Vevesca received fast track designation for the US Food and Drug Administration (FDA) in June 2000.
Genzyme plans to develop own
substrate deprivation therapy
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Source: Gauchers News March 2001.
© Copyright Gauchers Association 2001