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Print Edition StoriesThursday, January 21, 2010
Genzyme to make third attempt at FDA nod for Pompe disease drug
By Julie M. Donnelly
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Genzyme Corp. has announced that the U.S. Food and Drug Administration will issue a new decision on its drug for Pompe disease in June. The FDA first rejected the drug in March and then again in November. The second rejection was related to problems with the Allston-based manufacturing plant that produced it. The drug is currently approved in 40 other countries, where it is called Myozyme.
The company said in November that it would address the FDA’s concerns about the manufacturing processes at the Allston plant by adding internal controls, updating the filling and finishing capabilities in Allston and using contract manufacturers and Genzyme’s own Ireland manufacturing plant. Genzyme has since made several efforts on this front, including hiring a new senior vice president of global product quality and entering into an agreement with Lake Forest, Ill.-based Hospira Worldwide Inc. to provide fill and finish manufacturing services. Lumizyme is no longer being manufactured at the Allston plant.
Genzyme (Nasdaq: GENZ) was forced to shut down its Allston manufacturing facility for six weeks over the summer due to the discovery of a virus.
Genzyme last month reopened enrollment in a temporary access program for very sick patients, who can receive the drug before U.S. approval. The company has so far provided therapy free of charge to approximately 170 patients since 2007 through this program.
Pompe disease is a deficiency of a certain enzyme that helps the body break down glycogen, a complex carbohydrate that is converted to glucose for energy. Without the enzyme, glycogen builds up in the heart and other muscles.
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