BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) Q4 2022 Earnings Call Transcript

Hank Fuchs: Yes, to say a little bit more about VOXZOGO indications, we’ve covered this briefly, and there’s not really a ton more to update about other than to say that based on genetic data, the expectation is that a natural regulator of bone growth like VOXZOGO would be relevant in conditions beyond just that mutation that causes achondroplasia. Most — like to achondroplasia is a condition called hypochondroplasia, which affects the same gene but with different mutations that cause achondroplasia. And we’ve got some interesting preliminary data that an investigator has been working up at DC Children’s in an open-label investigator-sponsored trial. In addition, there are a number of other mutations, both in the same pathway or related pathways that also should be amenable to therapy with VOXZOGO, and he is now — Dr. Dauber has expanded his clinical trial to include patients with a variety of other mutations, including Noonans or NPR deficiency.

And one could imagine a conversation with regulators in which we’re talking about the eligibility criteria for a pivotal trial as either being directed at specific mutations or at a basket of mutations. And we plan to have further discussions with the agency about eligibility for trials as well as discussions about endpoints, duration, confirmatory requirements, et cetera. So, I think this process will unfold over the course of the year and we’ll keep you updated as we learn more in terms of the specifics about a program that could lead to expanded label claims for VOXZOGO. But at this point, we’re still at the beginning of the regulatory portion of the journey.

Operator: And our next question comes from Paul Matteis from Stifel.

Paul Matteis: Just two quick ones. I wanted to just clarify on JJ’s comments that you’re hoping to treat some ROCTAVIAN patients in Germany this quarter still. Is that contingent upon executing these other OBAs with the two important regional insurers? Maybe just clarify what has to happen for that to play out this quarter to start actually generating uptake? And then, more broadly on your annual ROCTAVIAN guidance, what’s your assumption on how back-end loaded the number might be, especially at kind of the mid- to high end of the range? And maybe comment just a little bit on your expectations for U.S. reimbursement and how long it will take for that to get on board?

JJ Bienaimé: I can start it and Jeff can. So, we already have, as we communicated for a while back, we already have one — in Germany that’s signed up. So, it’s one of the patients that is eligible after the competitive diagnostic is a patient that’s under — the umbrella under that fix fund, that patient could be treated any day. But hopefully, if we do end up signing another OBA or two other OBAs with the other fix funds, then it increases the probability that a patient will be treated this quarter. So with this, Jeff?

Jeff Ajer: The next part of the question was back-end loaded guidance and maybe…

Brian Mueller: Yes. I’ll handle it. Thanks, Jeff. Thanks, JJ and thanks, Paul, for the question. So first of all, maybe just a quick color comment on this ROCTAVIAN guide. It’s a wider range than you’ve seen in our other established products, but that’s because it’s a launch year in Europe. And as we touched on already on this call, the timing and of course, approval itself has some uncertainty in the U.S. So the way you can think about the guidance generally is the earlier we can get more of those German patients and then other European markets later in the year, fully on line, and then the earlier U.S. approval that we get that would push us towards the higher end of that guidance. And the longer or later that those things happen would push us towards the lower end.