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Genzyme Updates Data on Gaucher Drug

From Associated Press (February 11, 2010)

CAMBRIDGE, Mass. -- Genzyme Corp. said Thursday a potential Gaucher disease drug continued to show positive results after two years in a midstage study.

The company presented positive one-year study data on the drug in 2009, and it is now in late-stage, or Phase 3, development.

In the updated results, the drug candidate eliglustat tartrate, formerly Genz-112638, continued improvement in treating Gaucher, which is a rare condition that harms the body's ability to process fat. It can cause liver and neurological problems.

The midstage study involved 26 patients and 20 of those patients completed two years of treatment.

Genzyme, based in Cambridge, Mass., recently made several changes to its manufacturing leadership and operations following a year of problems.

In June, the company shut down its manufacturing plant in the Boston neighborhood of Allston to clean up viral contamination that had been slowing down production of Cerezyme and Fabrazyme. The virus was not harmful to people, but the shutdown cost the company millions in revenue. Production restarted in September.

Meanwhile, in November, the Food and Drug Administration said it found tiny particles of trash in drugs made by Genzyme, including steel, rubber and fiber. The agency recommended that doctors closely inspect vials of Cerezyme, Fabrazyme, Myozyme, Aldurazyme and Thyrogen.

Cerezyme also treats Gaucher disease and Fabrazyme treats Fabry disease, an inherited disorder caused by the buildup of a particular type of fat in the body's cells.

Shares of Genzyme rose 1 cent to $54.51 in morning trading.


Posted: February 2010