Seeking FDA Approval – Again

Genzyme hopes the FDA will approve their drug alglucosidase alfa (which is my ERT drug) on June 17,2010. The odds are mixed to whether or not this will finally happen as they have been seeking FDA approval for commercial use of this drug for years. They have experienced set-backs in the production of this drug at their manufacturing plant in Massachusetts.

Anticipating growing global demand, the company was moving production of Myozyme from Allston and a smaller facility in Framingham, Mass to a larger 4000 liter bioreactor scale in Geel, Belgium. However, upping production from 160 liter bioreactors to 2000L and 4000L scale-vats required that Genzyme submit supplemental Biologics License Application (BLA) to both FDA and European regulators, as the agencies insist that similar biologics produced in dissimilar bioreactors cannot be considered identical products, due to differences in the carbohydrate structures of the molecules in the so-called brews. Confused? That’s OK: the bigger tubs of alglucosidase alfa will continue to be sold under the Myozyme brand in Europe and under the Lumizyme title stateside. — BNET.Com, November 19, 2009, “FDA Delays Genzyme’s Lumizyme, Manufacturing Problems Blamed” *

What this means for Pompe patients is that, if it is approved those not currently receiving the drug through an ATAP study should be able to get it. And for those of us in an ATAP study, the study will end and we’ll join everyone else in the commercial use of the drug – and having to pay for it. Let the insurance battles begin!

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