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

Jeff Ajer: For VOXZOGO launch trajectory, I think the question was about what are you seeing in terms of age distribution and uptake. You specifically mentioned Japan. And so I would comment that Japan indeed has been — we’ve seen rapid uptake in Japan since we got reimbursement approval last August. Remember, Japan is an established market for treating achondroplasia. So, it’s unique in that aspect. And we’ve been fortunate to tap into that established care model for achondroplasia in Japan. In addition, Japan is a — VOXZOGO is approved for all aged patients in Japan. There is no younger limit. And indeed, we’ve seen a lot of interest and uptake in the younger patient segments in Japan. In other markets to the extent that we have visibility into this data, which is somewhat limited in Europe because of data privacy regulations.

A year ago, I was commenting on the diversity of the age segments that we’re seeing patients start at. We’re seeing very young patients and even patients that were key majors starting treatment. Today, I would say the data looks like it’s concentrating towards younger patients starting therapy, which we think is a good dynamic and bodes well if we can get approval for younger age segments in Europe and the United States. And finally, a note just a little bit of a plug, what the data that we have would suggest really high compliance with patients being treated so far, which is encouraging.

Hank Fuchs: And on your question on 331, there’s really no further update to give. I mean, in the past, what we talked about at JPMorgan, we have an individual patient who is beginning to express, moving into the normal range. But there’s a lot of variability in week-to-week visits, and we have another patient to treat before we can make a determination about whether this dose level appears good enough or we want to expand to another dose. So encouraging start, but stay tuned for more.

Operator: And our next question comes from Matthew Harrison from Morgan Stanley.

Matthew Harrison: I guess just one follow-up on VOXZOGO. Jeff, can you just maybe talk in a little bit more detail about how you’re thinking about the contributions to guidance this year? Is this mainly geographic expansion and new patient adds? Or are you also expecting as part of guidance to see younger patients be added into the label and therefore, drive uptake? And then, just a corollary to that, Hank, can you just talk about some of the things you’re thinking about in terms of other indications for VOXZOGO and sort of the progress there? Thanks very much.

Jeff Ajer: Hi Matt, I’ll start off and then turn it over to Hank. The guidance for VOXZOGO reflects the current trajectory that we’re on with respect to new patient additions and the base of patients remaining pretty compliant and contributing to revenue this year. So, we’re following the overall trajectory of that launch. It is true that a global launch is really the sum of individual markets launching. So, it’s a very dynamic situation. We’re in 32 markets now including all of our strategic markets. And like I mentioned a minute ago, we’ve gotten a lot of rapid uptake in Japan. That’s a big strategic market for us, and we’re relatively early into that launch cycle. Other big markets like Germany and the United States, we’ve been in longer, but we’ve still got plenty of room for growth.

We do anticipate that we would see the benefit of having expanded labels this year, but that’s not fundamental to the guidance range, I would say. That would be helpful, but not fundamental.

JJ Bienaimé: Yes, because if we do get approval regarding age extension, it’s not going to happen before probably Q4. So it’s not going to have a major impact on our revenue this year, but it will be good for €˜24. Hank?