Taliglucerase alpha was up for approval two years ago, but the decision was delayed because the FDA wanted more information on the manufacturing process for the drug, which is made in plant cells rather than the more traditional mammalian cells. Eventually, the FDA rejected the drug, requesting clinical data on the switchover trial and other information about how it tests the drug to make sure it's been produced correctly.
I hate these types of binary events. There are too many unknowns with manufacturing issues to feel confident about an approval. Discovery Laboratories
And it's not as if the FDA is likely to be lenient just because Gaucher disease is rare. The FDA turned down ViroPharma's
Protalix says it's answered all the FDA's questions, and I have no reason to think otherwise. But investors have very little insight into what the FDA is thinking about the manufacturing issues. I hope taliglucerase alpha is approved, but given the unknowns, I don't see how it's worth placing a big bet on the agency's decision.