Manufacturing woes plague launch of Myozyme

Genzyme has watched its share price slide recently as it struggles to gain FDA approval for a new manufacturing facility to make Myozyme, a super-expensive enzyme-replacement therapy for Pompe disease. Genzyme says that it's urging patients to get doses through the unapproved facility that produces the therapy for experimental uses. But that source is free, instead of the $300,000-plus a year that the drug costs on the open market. The Wall Street Journal cites a knowledgeable source who says that Genzyme's problem is its inability to produce the therapy in the same version made for clinical trials. Genzyme plans to resubmit its application later this year--provided it can make matching therapies. About 10,000 people in the world have Pompe disease, and about 700 are taking Myozyme.

- read the report [1] from The Wall Street Journal (sub. req.)

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