Genzyme reports encouraging data for Gaucher disease drug candidate

Genzyme, the Cambridge biotechnology division of French drug maker Sanofi SA, reported positive data for an experimental drug for Gaucher disease, a rare genetic disorder that can cause swelling of the liver and spleen.

Genzyme has an existing Gaucher disease drug on the market called Cerezyme. In 2008, when Genzyme was still a public company, Cerezyme accounted for $1.24 billion in revenues, or 30 percent of total company revenues. (Cerezyme revenues in 2009 and 2010 were hurt by production problems at the company’s facility in Allston. Sanofi bought Genzyme for $20.1 billion in 2011, and Genzyme no longer breaks out drug revenues.)

Cerezyme is delivered by infusion, once every other week, and the infusion process can take between one and two hours.

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The new Gaucher disease drug candidate is taken in pill form so it has the potential to be a more convenient option for some Gaucher disease patients, Genzyme said in a press release.

That drug candidate, eliglustat tartrate, is in its final phase of clinical testing. If all goes well, the next step would be for Genzyme to apply to regulators for approval to market the drug.

People with Gaucher disease are deficient in an enzyme that breaks down a certain type of fat molecule. As a result, lipid engorged cells (called Gaucher cells) amass in different parts of the body, primarily the spleen, liver, and bone marrow. Accumulation of Gaucher cells may cause spleen and liver enlargement, anemia, excessive bleeding and bruising, and bone disease.

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