Jean-Jacques Bienaime: Yeah, in addition to the federal building up, though, I mean, it is possible, I think that was your question, can we treat some US patients by year end [ph] I would say, yes, it is possible. We have three US patients who have now cleared all ROCTAVIAN screens for treatment, like the AB5, liver scan, liver health, and all that. So, the timing of their treatment isn’t guaranteed in ’23, but they have completed all eligibility testing for treatment, so it will very likely occur in the not too distant future.
Operator: Your next question comes from the line of Robyn Karnauskas of Truist. Your line is open.
Robyn Karnauskas: Great. I have three quick questions. Number one, JJ, you have mentored so many people on the street, so appreciative. Is Hank retiring too, because we will have a panic attack, so like, would you like an announcement on that call? Is Tracy getting a promotion? That’s our first question. The second question would be, ROCTAVIAN, there was such a focus, and you misguided this year on the outcome of what you could achieve. Do you think in 2024 you can do better? And then I think on VOXZOGO, I mean, that came away from our analyst meeting as like the big takeaway. So, how do you think about letting analysts understand the opportunity there and the timelines for that? And I’ll go back to Tracy’s promotion. Is Hank leaving? I’ll leave it at that.
Jean-Jacques Bienaime: Thank you, Robyn. I mean, I think there was a previous question from, I think, from Salveen, and we had a detailed answer from an analyst from Geoff. Indeed, it’s taken longer than we anticipated to get the – to connect the patient with the treatment centers, to get the whole system wired. So, again, big picture, when you have such a big ticket item like ROCTAVIAN, $3 million, whack, it attracts a lot of people. So, essentially, it’s a big pie that attracts a lot of players that you don’t get involved in the decision-making because they all kind of want a piece of the action. Just so Jeff mentioned the 340B. 340B is 20, I mean, all basically, all hemophilia patients in the US, statutory-wise, are 340B patients.
When you’re talking 20%, 340B discount with $3 million whack drug, you’re talking about $600,000. As you can understand, many people get interested in that pie and want a slice of it, which just slows things down. But eventually, long-term, it’s actually good news that it will be taken care of. So, the fact that the takeoff has been delayed, to me and to us at BioMarin has no impact on the future potential of these drugs. It’s taking longer than planned. It took longer – I’m sorry, when the first time we got a product approved in Europe before the US, and ROCTAVIAN got approved in Germany, and based on our previous experiences, you know, we thought we could be able to treat some patients during the free pricing period in Germany, which is slowly disappearing, didn’t happen.
Then we are basically, you know, very, very close to a final deal with the German authorities, which generally takes about 12 to 13 months. So, in this respect, we’re not that far behind versus the European approval. So, again, we believe ROCTAVIAN has great potential. We believe ROCTAVIAN would be a significant contributor in 2024 and beyond. Obviously, not as much as VOXZOGO, which is turning into a very, very large drug based on our Q3 results, not to say Q4, and the fact that, thanks to the efforts of our drug supply team, all the constraints in terms of packaging that we had and sealed finish that we had this year are starting to go away and will be much more substantial. We have much more substantial capacity in Q1 and Q2, and then be free of all capacity constraints by the middle of the year.
So, I hope that answers your question, and I’ll get back to you regarding Tracy.
Operator: Your next question comes from the line of Joseph Schwartz of Leerink Partners. Your line is open.
Joseph Schwartz: Great. Thanks very much, and best wishes to JJ. That’s really the end of an era. So, I guess I was wondering if you can help us understand what kinds of initiatives you’ve undertaken to address the administrative delays and site readiness issues, and, you know, what kinds of success have you had where you have implemented these things, and what initiatives do you have left to implement still to support ROCTAVIAN’s growth? Any more color there would be very helpful.
Jeffrey Ajer: Thanks, Joe, for your question. I would characterize the answer to that as less specific initiatives and more just, you know, the nuts and bolts and mechanics of selling, of providing scientific support to HTCs, the physicians, patient advocacy groups, including local chapter events, all the things that help them through those little steps and lots of little decisions, including the big decision of affirmatively making the decision to treat. And as Hank and I have discussed just this week, we’re really focused on those small tactical, on the ground elements with our teams to just keep those things moving through the various little challenges that are popping up. So, it’s more about dealing with those little things, getting to the other side of them, many of which are one-time events, and less about introducing new initiatives.
Joseph Schwartz: Thank you.
Jean-Jacques Bienaime: And if I may ask you, regarding Germany, again, in Europe, you know, patients don’t pay for drugs. They don’t pay either for doctor’s visits, so they don’t know the price of a drug. But what, so, the key thing in Europe is to have official government reimbursement for your drug. That’s about to happen in Germany and Italy. We basically, as we’ve said, we have an agreement on the price in Germany already. Now we need to have this price published, which should occur over the next few weeks. Once that happens, there is no barrier whatsoever left, you know, in Germany, because 60 patients are 85 negative in Germany. It’s very likely that the vast majority of them will be treated. Obviously, most of them next year, because there are so few weeks left in the year because of the holiday season is coming up, you know, pretty soon, and the fact that the price probably won’t be published for another, three or four weeks.
Operator: Your next question comes from the line of Chris Raymond of Piper Sandler. Your line is open.
Chris Raymond: Thanks, and congrats from us, too, to JJ, and sorry to see you leave, JJ, and really enjoyed working with you all these years. I’ve got two questions, maybe one a commercial one, maybe for Jeff, and then one for Hank, if that’s okay. So, maybe just on VOXZOGO, Jeff, just on that supply constraint issue, maybe could you talk a little bit about the triage process that you’ve instituted with new patient starts? I guess, I’m just kind of wondering what kind of retention programs do you have in place for those new patients who want to be on therapy but can’t. And can you give us a sense of that number now, and where you expect it to grow to, you know, when you get back up to supply mid-next year and start to treat those patients?
And then, maybe on the R&D side, Hank, just noticing back at your R&D day, which was just in September, I think your cardiomyopathy programs, I think 293 was supposed to have its initial data proof of concept in 2025, and I think 365 was the year after. It looks like those have been pushed a year. It’s only been a few weeks since your R&D day. Maybe, is there a sense of what’s happening there? Thanks.
