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A UK-based key opinion leader (KOL) provides insights into prescribing habits, key marketed drugs, and late-phase pipeline therapies for hemophilia, including alternative coagulation promoters and gene therapies. Unmet needs are also discussed. The main marketed products covered include Hemlibra, Advate, BeneFIX, and Eloctate, and key pipeline assets highlighted are valoctocogene roxaparvovec (valrox), fitusiran, and concizumab.
Highlights
OK, it’s a bit different for A and B. If it’s B, the extended half-life products have a dramatic effect, so that is to say they produce a very dramatic prolongation of their half-life, so we’ve moved all of our severe onto Alprolix actually, which is one of the extended half-life Factor IX preparations. For hemophilia A, the extended half-life was less dramatic, and so there were some restrictions from NHSE on the extent to which we could use it. So, while we’ve moved several people onto Elocta, and in fact the restrictions have loosened a bit recently because Sobi have reduced the price of Elocta, and we have moved a few people over, we still have quite a few people on what we might call the conventional standard half-life products like Advate, NovoEight, and ReFacto.
For hemophilia B first, I expect Alprolix to gain more of the market, but I think that’s happening already, and I think that will continue. There isn’t any real competitor on the horizon, there’s a modified Factor IX molecule that’s more potent, and will be given subcutaneously, that may come into license in the next few years, actually I think it’s going to have trouble. But in the Factor VIII market, it’s more complicated because you have Hemlibra coming in, and Hemlibra is going to take progressively more and more of the market I think because of the characteristics of the trial, that it’s a subcutaneous injection once a fortnight, and pretty good efficacy. So, apart from people who need particularly high levels, or who have very energetic lifestyles, or are actually very happy with what they’re doing, most people are not particularly happy about injecting themselves intravenously every other day, I think – although it may not have a big splurge, because I think there’s a lot of inertia in the system, I think it will progressively eat into the market, and the use of certainly standard half-life, and then also EHLs, except the fact that there are other EHLs coming through, like BIVV001, which are going to be a challenge for Hemlibra.
Well, the big advantages [of Hemlibra] are that it’s administered subcutaneously, which is very easy compared to intravenous, and it’s given every fortnight generally speaking, which is much less frequently than every second or third day that conventional products are given. So, that makes life a lot easier for people, and it’s very attractive, as I said people don’t like injecting themselves that much. So, I think that’s a big plus. It gives you a very steady level of coagulant activity compared to the ups and downs of conventional Factor VIII molecules, which for many people is an advantage because it seems to be sat at a reasonably high level, maybe 10–12%, and yet, for some people the peaks are important, the disadvantage is that you don’t get very high levels. So, if you wanted to give yourself a shot of something before you go and play football, Hemlibra is not the stuff to use. It would be a standard half-life product that would probably be just as good as anything, or maybe an EHL. So, that’s its disadvantage really, and that’s about it actually. The antibodies seem to be very, very rare, so it’s not a problem there, and if you do get them you just go back to Factor VIII. No toxic or allergic reactions very much really. It’s just a question of whether you need a higher level. So, for people with inhibitors it’s a bit more complicated, but not really.
A UK-based key opinion leader (KOL) provides insights into prescribing habits, key marketed drugs, and late-phase pipeline therapies for hemophilia, including alternative coagulation promoters and gene therapies. Unmet needs are also discussed. The main marketed products covered include Hemlibra, Advate, BeneFIX, and Eloctate, and key pipeline assets highlighted are valoctocogene roxaparvovec (valrox), fitusiran, and concizumab.
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