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

And specific to your question, you’re exactly right that given the uptake trends given both those European and U.S. dynamics would suggest that it is back ended and you’d expect the larger portion of the revenues to come in the second half of the year.

Jeff Ajer: And then back to the third part of the question about U.S. reimbursement. And you’ve seen this from BioMarin before, right? When we get approvals in the U.S. and we’re able to — for high value and high value-added therapies, we’re able to get reimbursement going in the United States pretty quickly. That’s based on a couple of dynamics. One is we’ve got an experienced team out there. And we know how to get through the medical exception process while we’re waiting for coverage policies to be issued. And for ROCTAVIAN anyway, there’s always going to be — are likely to be always prior authorization process steps there that we know how to navigate. The U.S. system is highly diversified. That’s both a challenge and an opportunity for us relative to going through federal reimbursement process, like we are in Germany and France and Italy, where at least in France and Italy, you have to get all the way through the process before you can treat patients and that takes a year.

Relative to the U.S., I mentioned in the prepared remarks, the warranty is a key aspect of facilitating rapid patient uptake. The warranty is something we offer with purchase. It’s an outcomes-based agreement. It covers risk for insurers. We offer it with the purchase of ROCTAVIAN, means we — there’s no negotiating the terms, and we don’t have to negotiate and get to contract signature with lawyers involved in rounds of review and that sort of thing. So the warranty is an essential element. And finally, pricing correctly to give U.S. payers a financial incentive to support ROCTAVIAN is important. And to that end, we had the final report from ICER in the United States. It said ROCTAVIAN is a dominant choice relative to Hemlibra. And they previously concluded that ROCTAVIAN was a dominant choice relative to Factor VIII replacement therapy at a presumed price of $2.5 million.

So that gives us a lot to work with in terms of lining up price and the financial incentives. Thanks.

Operator: Our next question comes from Tim Lugo from William Blair.

Lachlan Hanbury-Brown: This is Lachlan on for Tim. Hank, I understand you can’t give specifics. But can you just confirm if you have sort of clear alignment with the FDA on the requirements for devoting VOXZOGO’s accelerated approval to approval — full approval and potentially any guidance on like when you might be able to share more details around that? And then also, as we look towards the steroid prophylaxis study for ROCTAVIAN in Q2, can you just maybe talk about like what you need to see there to, I guess, feel like you’re confident that the steroid prophylaxis either does or doesn’t really impact therapy?