LL schreef op 23 december 2021 10:36:
Dit is het laatste wat we hebben kunnen vernemen m.b.t. Pompe (uit de analisten cc)
I’ve got 2 or 3. I mean, first of all, I was wondering if you could give us a timing on submission of the IND application in Pompe? Secondly, I was wondering -- I mean, I’ve noticed over that a number of gene therapy-based approaches are now in trials on Pompe. And I was just wondering if you could comment on how your own approach stacks up against those? And thirdly, just on the marketing costs. I think year-on-year, you’ve had a $7.7 million increase. I was just wondering if you could split out how much of that increase relates to leniolisib and other items and how much relates to RUCONEST?
Sijmen de Vries
Yes. Shall I answer -- the first one first then, Simon, about Pompe. Pompe’s disease, we’ve taken -- of course, our product -- our development is hampered significantly at this point in time by unavailability of materials necessary for manufacturing. That is a problem that we have in the pharmaceutical supply chains widely, and we are no exception for that, unfortunately.
Secondly, so I can’t give you a proper date for the IND. With regards to the uncontinued medical -- unmet medical need. We’ve just done a deep dive into this indication. And we concluded that the unmet medical need is there -- is still there, and we expect it also to continue to be there. Also, because gene therapy might have some additional hurdles in such a lysosomal storage disease as Pompe compared to many of the other diseases.
So the answer is we continue to be interested in Pompe. We’re significantly delayed because of the COVID issues with manufacturing, and we have no data at the moment for an IND, therefore, as these supply chain disruptions are widely spread and COVID is not going back and not retreating, in fact. So it’s difficult to say at the moment. That’s my first question.