Genzyme starts shipments of drugs for two rare disorders

02 Dec 2009

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Even as drug giant Pfizer announced its pact with Protalix to develop and commercialise treatment for genetic metabolic disorder Gaucher disease (See: Pfizer inks $115 million deal with Israel's Protalix for treatment of Gaucher's disease),  Genzyme Corporation announced that it has begun shipping vials of newly produced Gasucher's teatment Cerezyme (imiglucerase for injection).

The initial Cerezyme shipments will provide uninterrupted treatment for the most vulnerable patients under the current supply conservation guidelines. By the end of this month, Genzyme will expand shipments of Cerezyme to patients worldwide who have experienced interruptions in their treatment this year.

Genzyme  expects that it can meet anticipated worldwide demand and allow all patients to return to their normal infusion and dosing regimen during the first quarter of next year. Patients have been asked to contact their treating physicians for information on the timing and process to re-start their infusion programme.

Gaucher disease is an inherited condition estimated to be affecting fewer than 10,000 people worldwide. People with Gaucher disease do not have enough of an enzyme, ?-glucosidase (glucocerebrosidase), to break 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, bone disease and a number of other signs and symptoms.

Genzyme is also preparing to ship vials of newly produced Fabrazyme (agalsidase beta).

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