Fabry patients got more bad news yesterday -- as if it weren't bad enough already, given shortages of Genzyme's Fabrazyme in the US due to that company's well chronicled manufacturing problems. In fact, last week an Idaho woman filed a lawsuit against Genzyme, charging that a long-standing shortage of its Fabrazyme treatment for Fabry disease led to the death of her husband two years ago. He would still be living, she claims, if the biotech had not experienced severe and persistent quality control problems that led to a subsequent rationing program.
On the heels of this, Shire had been encouraged by patients and the FDA itelf to file a BLA to get its Fabry drug Replagel approved in the US. Now, Shire says that the FDA has told them they will require more clinical data despite the fact that Replagal has been on the European market for more than 10 years.
However, Shire says that recent meetings with the FDA indicated additional controlled trials will be required for approval, although safety was not an issue. This comes after the drugmaker last November filed its application with the agency, anticipating a speedy review. However, the added trials will cause “a significant delay” and approval would not occur until “the distant future.”
Analysts called the move "a surprise setback for Shire," according to Bloomberg. A new trial, they added, "would take at least two years and is probably not economically justifiable." All in all, not good news for Fabry patients. See Fierce Biotech, Pharmalot, and PharmaGossip.
The only one who may be cheered by this news is Genzyme and its investors as its new US manufacturing facility to produce Fabrazyme was recently approved. The company can now go back into large scale production. One might wonder about the timing of the FDA decision given that set of circumstances.
Posted by Bruce Lehr Mar 16th 2012.
