Replies to post #162926 on Biotech Values

[ghmm]

PLX / KMDA:

I thought being a recombinant product would convey some safety benefits but perhaps that wouldn't enough to break orphan exclusivity?

[genisi]

PLX, KMDA, and orphan exclusivity

When the CHMP recommend against marketing approval for PLX's enzyme in Gaucher (I was wrongly thinking PLX had a very good chance of bypassing Vpriv EU exclusivity then), it concluded that "Elelyso is similar to Vpriv, as they are both enzyme replacement therapies that work in the same way." Didn't matter how the enzymes were produced or their different glycosylation. Things could be quite different in court in patent litigation (I'm thinking of Enbrel).
I don't know what strategy PLX would pursue in the AAT case if KMDA gets orphan exclusivity but they are holding an analyst day here tomorrow, so perhaps we'll have answers.

[DewDiligence]

PLX/KMDA—…how similar the competing enzymes have to be before they are judged to be "the same chemical entity" and thus disallowed under orphan exclusivity.

The criteria may vary to some degree according to jurisdiction. EU orphan exclusivity runs for 10 years, while FDA orphan exclusivity runs for 7 years; on this basis alone, one would expect some differences in how the regs are applied.

[iwfal]

Orphan drug exclusivity - how different does a new drug have to be to break the definition of 'exclusivity'? The FDA just gave Teva orphan drug exclusivity for a deuterated version of a previous drug. So not very.

http://tinyurl.com/y8m68a9w