Gena Wang: I wanted to ask about ROCTAVIAN again. For the 18 patients that completed the antibody testing, how many of these were eligible? Also in Europe, since now you’re pursuing directly with GKV without pursuing Outcomes Based Agreement with the sub-insurance, does that mean your price will be much lower than EUR 1.5 million that you previously discussed? And then lastly, very quickly regarding the U.S., can you discuss on hemophilia A patient under 340B program? And how does that mechanism impact the ROCTAVIAN initial launch?
Jeffrey Ajer: Thank you, Gena, for those questions. Let me start with the 18 CDx tested and percentage that are eligible. So first thing to note is CDx testing is one eligibility test, liver health is the second. Relative to the AAV5 seropositive or negative status, the best thing that we can do to guide our expectations on that is the publication from our seroprevalence study in hemophilia A. And in that study, which was published, the overall seropositivity rate for AAV5 in Germany was 35%. So that’s guiding our expectations on the percent of patients that would be eligible based on AAV5 negativity. Relative to the GKV price negotiations and what we had guided to as net price of about EUR 1.5 million, which was last fall. We think it is likely based on what the GKV is statutorily allowed to do, we think it’s likely that they will not be putting into the agreement and outcome space component or a pay overtime component, but nothing is certain until we get done with that process.
But you’re right. In that particular case, we think that the price negotiations with GKV would incorporate all of the aspects of value of ROCTAVIAN, including durability, for example. So we think that the final price would model in how GKV is looking at durability of ROCTAVIAN over time. And then relative to the 340B question in the United States, in our script remarks, we mentioned that we’re committed to this HTC model of treatment and follow-up hemophilia treatment centers or HCCs in the United States, our granted 340B eligibility, that’s a way of kind of funding the work — the important work that they do to care for hemophilia patients. So we think all or substantially all of our revenue in the United States would be subject to the 340B discount.
Jean-Jacques Bienaime: What’s your what that’s going to do to the launch some respects, it’s positive for us. .
Jeffrey Ajer: Yes. We think that’s very positive. So as I mentioned, the reason for being for HTC is to have access to the 340B discount is to have a source of revenue to fund their important operations. And as ROCTAVIAN would likely be eligible for those 340B discounts for all or essentially all of their patients, that’s revenue that would accrue to those to fund their operations, and that revenue would happen at the time of treatment versus, for example, revenue that they might be getting from supplying factor replacement products which they would see over time. And by the way, an important point to note is the HCC see a revenue component from factor replacement therapy only for a small proportion of their patients on average. So we think that this is actually maybe a motivating factor for treatment with ROCTAVIAN in the U.S.
Operator: And our next question comes from Tim Lugo from William Blair.
Timothy Lugo: You mentioned VOXZOGO is on its way to becoming the first blockbuster for the company. Will these other non-achondroplasia indications add to the market? And kind of how much — and can you also update us maybe I missed this on converting the accelerated approval to a full approval?
Jean-Jacques Bienaime: Tim, I’ll start, and then Jeff can provide more echo. I mean as we made in the prepared remarks, I think in Jeff’s remarks, we only have penetrated about less than 10% of the market right now for VOXZOGO achondroplasia. So obviously, we can get past and we are on a run rate already of $450 million or so. So obviously, in the market alone, there’s a lot of room to grow. And we could easily pass the $1 billion in revenues a lot. So obviously, other indications are going to make it even larger, but that’s kind of my first comment to your question. Jeff?
Jeffrey Ajer: Nothing to add there. Thanks, JJ. And then there was a question of accelerated the full approval rate.
Henry Fuchs: Nothing new to report there. It’s published post marketing requirement and the specifics of the timing for that, we’ve kept those relatively proprietary. So stay tuned.
Operator: And our next question comes from Paul Matteis from Stifel.
Paul Matteis: Surprised it took a long time to get a question on the FDA with ROCTAVIAN, but I thought I’d just throw it in there. You should be only a handful of weeks away from labeling discussions. Just curious if you could update us on the cadence of your discussions and how everything is going? And then if you do get approval in June, do you think these reimbursement warranties could be agreed upon and in place quickly enough to generate meaningful revenues for ROCTAVIAN in the U.S. in 3Q?
Henry Fuchs: Thanks, Paul, for the question. I won’t get into the specifics of the back and forth. But I would say that as a general matter. The things that we expect to be having at the stage of the review appear to be happening. And I think that gives us optimism. I think we’ve also expressed some caution there because we don’t have perfect visibility into everything going on in the agency and exactly where senior management and the people who signed the letters are, but we’re optimistic.
Jeffrey Ajer: Relative to the warranty, Paul, the nature of the warranty is that it is uniform. It is nonnegotiable. It is available to all purchasers. And essentially, we don’t have to negotiate the purchaser essentially gets that warranty with purchase. So the timing is relatively trivial.
Jean-Jacques Bienaime: No timing.
Jeffrey Ajer: No timing, yes.
Jean-Jacques Bienaime: No delays
Jeffrey Ajer: No.
Operator: And our next question comes from Robyn Karnauskas from Truist Securities.
Robyn Karnauskas: So just clarify, for ROCTAVIAN, since you’re not doing OBAs anymore, can you give us some sense of what price we should be using since that’s been the fixation of a lot of us for Europe and how to think about that? And second for VOXZOGO, you’re averaging now like 400 patients a quarter. You mentioned a lot of expansion, new indications, geographic. Should we think of it more consistent or choppy?
Jeffrey Ajer: Okay. Let me start off with the price component. We’ve guided roughly last fall to net price expectations in Europe. How we get there is different, whether there’s an Outcomes Based Agreement or no Outcomes Based Agreement, and that’s figured into the value of the durability of which is figured into the value of the upfront price. But the over — where we wind up should not be materially different from either path.
