Wednesday, September 24, 2008

Genzyme’s Myozome treatment for Pompe disease awaits FDA approval

By Mass High Tech Staff

Genzyme Corp. has announced that Myozyme, its treatment for the gradually debilitating genetic disorder Pompe disease, is awaiting review from the U.S. Food and Drug Administration.

On October 21, the FDA Endocrinologic and Metabolic Drugs Advisory Committee will meet to determine whether Genzyme’s biologic license application may extend the use of alglucosidase alfa beyond its current approval at the early onset, 160L bioreactor level. The treatment for adults requires new FDA approval, with a new biologic license application, due to its change in carbohydrate structure made at the 2000L bioreactor scale.
 
The meeting consists of a private discussion of Genzyme’s manufacturing data, as well as a public meeting to review the results from the company’s treatment study of Myozome used in late-onset cases of Pompe disease.

Earlier this month, Genzyme (Nasdaq: GENZ) announced that it had issued a recall of a single lot of its anti-rejection drug, thymoglobulin, due to the fact that it may not be as stable as it should. The FDA said Genzyme had problems with, among other things, its purified water system and quality control unit at the plant. The potential problems could lead to impurities in the drug if the company does not take action, the FDA said.

With 11,000 employees worldwide, Genzyme reported a 2007 profit of $481 million on revenue of $3.8 billion.