Biotech Values

[iwfal]

PLX -

Yes, if KMDA gets approved with their inhaled enzyme, PLX will have to try and break KMDA's orphan exclusivity and I don't think chances are very good (recall they tried this with Shire's orphan exclusivity in EU in Gaucher and failed.

It would be interesting to watch the regulatory and court decisions to see how similar the competing enzymes have to be before they are judged to be "the same chemical entity" and thus disallowed under orphan exclusivity.

1) Same active-in-cell ingredient is the only thing that matters? - regardless of delivery mechanism (which is, or course, a large part of the trick in enzyme treatments)?


2) Will different glycosylation count as being different? ...

Given the likely uncertainty/risk I am surprised that companies aren't being more careful to try to be at least a little different on the final enzyme - e.g. start with a chimp variant.