US Food and Drug Administration (FDA) approval is now imminent, according to Genzyme, which would draw a line under a challenging 10 months for the company’s Myozyme.
In an effort to scale up production of Myozyme Genzyme built a facility housing a 2000L bioreactor, which offered considerably more capacity than the 160L site.
However, the FDA ruled that in the process of scaling up from 160L to 2000L the product had changed sufficiently to require a new Biologics License Application (BLA).
This was because of concerns about the glycosylation of the protein, which the FDA believed had been altered to such an extent in the 2000L scale production process that the product is materially different from the previous version.
Genzyme suffered as a result of the ruling, with capacity going unused and Myozyme sales not increasing to the extent predicted. However, having got through this difficult period Genzyme believes it is well placed for the next 12 months.
Henri Termeer, Genzyme’s chairman and CEO, said: “We had an excellent year last year and exceeded our earnings expectations despite the economic environment and the challenges we faced with Myozyme.
“We delivered on our financial objectives but did not ignore our future. Genzyme is stronger today than it was a year ago, and we feel confident about our future.”
Genzyme is also hopeful that it will gain approval for the 4000L perfusion reactors that it has built in Belgium, which will be another factor in driving Myozyme sales over the coming year.