RRR schreef op 30 november 2012 09:56:
Views towards prophylaxis
While we look for Ruconest to gain traction in the acute HAE setting, we believe an area of significant upside
potential exists in the prophylaxis market and that Ruconest could pose a significant threat to ViroPharma’s Cinryze.
Considering the profile of more chronic use, Ruconest could look again to differentiate itself on both the efficacy and
safety standpoints. To this end, evidence to date supports the potential of dosing even higher then 50 U/kg with
Ruconest, whereas we perceive the dosing of Cinryze to be capped to potential safety issues such as
thromboembolic events at higher concentrations. On the safety point, as mentioned above, after >1,000
administrations, Ruconest has not seen any thromboembolic events whereas Cinryze and Berinert are known to have
that risk (in clinical studies Cinryze saw 3.4% incidence). Though not officially proven yet, we attribute these safety
differences primarily to the fact that Ruconest is a purified recombinant C1 inhibitor, whereas Cinryze and Berinert
are plasma derived proteins whose formulations contain 20-25% “impurities” many of which are unknown (beyond
infectious disease testing that is part of purification process).
While Santarus/Pharming’s focus is not currently (publicly) on the prophylaxis opportunity, we believe that
background activities are already
in full force in prepping for planned FDA discussions. In considering a potential
Phase III, we believe that the study would be in the 30-60 patient range and cost in the €5-10 million range. It is
possible, though remains to be seen, that Pharming and Santarus would split the cost of this study 50/50.
En ineens bedenk ik...daarom had/heeft men dus geld nodig... :-)