Pharmaceutical Business review

Genzyme Gaucher disease drug trials show positive results

<p>The company said that as a result of the encouraging results seen in trials of the drug to date, it intends to meet with regulatory agencies in the US and Europe in the coming weeks to discuss an expedited development strategy. <br /><br />Genzyme reported that initial observations from phase II trials suggest that the drug, Genz-112638, may have a rapid and meaningful impact on important clinical endpoints, including reductions in spleen and liver volume, and an increase in platelet counts and hemoglobin concentration.<br /><br />The company added that the only drug-related negative effects seen in patients enrolled to date have been mild and transient. There has, however, been one possibly related serious adverse event that is currently being investigated.<br /><br />The company said that if the early improvements continue and are observed in other patients enrolled in the trial, Genz-112638 may represent a promising approach to treating patients with Gaucher disease. Genzyme&#0039;s full trial results will be available in mid-2008.<br /><br />David Meeker president of the company&#0039;s lysosomal storage disorder business, said: We have set a very high bar for ourselves in trying to develop a convenient oral therapy that can provide a safe and effective choice for patients. We look forward to developing this molecule further and exploring the role it may play in the treatment of patients with Gaucher disease.<br /><br />Gaucher disease is caused by a deficiency of the enzyme glucocerebrosidase, leading to an accumulation of its substrate, the fatty substance glucocerebroside. Fatty material can collect in the spleen, liver, kidneys, lungs, brain and bone marrow. Symptoms may include enlarged spleen and liver, liver malfunction, skeletal disorders and bone lesions.</p>