Ultragenyx Pharmaceutical Inc. (RARE) Earnings Call Transcript & Summary

Ladies and gentlemen, thank you for standing by, and welcome to the Dojolvi U.S. FDA approval call. [Operator Instructions] I would now like to hand the conference over to your speaker, Ms. Danielle Keatley. Ms. Keatley, the floor is yours.

Thank you. Good afternoon, and welcome to the Ultragenyx conference call to discuss today's U.S. FDA approval of Dojolvi. We've issued a press release detailing the approval, which you can find on our website at ultragenyx.com. I'm Danielle Keatley, Senior Director of Investor Relations. Joining me on this call are Emil Kakkis, Chief Executive Officer and President; Erik Harris, Chief Commercial Officer; and Camille Bedrosian, Chief Medical Officer. I'd like to remind investors that this call will include forward-looking statements within the meaning of the safe harbor provisions of the Private Securities Litigation Reform Act of 1995 including, but not limited to, types of statements identified as forward-looking in quarterly report on Form 10-Q that was filed on May 7, 2020, and our subsequent periodic reports filed with the SEC, which will be available on our website in the Investors section. These forward-looking statements represent our views only as of the date of this call and involve substantial risks and uncertainties, including many that are beyond our control. Please note that actual results could differ materially from those projected in any forward-looking statements. For a further description of the risks and uncertainties that could cause actual results to differ materially from those expressed in the forward-looking statements as well as risks relating to our business, see our periodic reports filed with the SEC. I'll now turn the call over to Emil.

Good afternoon, everyone, and thank you for joining us. Earlier today, we received FDA approval for Dojolvi for the treatment of children and adults with long-chain fatty acid oxidation disorders or LC-FAOD. This group of rare disease includes 6 distinct enzyme deficiencies. We believe that Dojolvi will be an important therapy for these patients who, up until this time, had no FDA-approved treatment option. The vision for Dojolvi began more than 20 years ago with the work of Dr. Charles Roe, who at the time was at the Baylor Research Institute in Dallas, Texas. Dr. Roe first recognized and established that there was a significant unmet need for the treatment of LC-FAOD, which was not being well-managed biochemically with the options available at that time. His understanding of the important fuel cell in the body that is required for energy creation, called the Krebs cycle, allowed him to identify triheptanoin as a potential replacement for this deficiency in LC-FAOD. His decade of clinical research demonstrated the unique properties of triheptanoin, and he published several articles on its potential to treat the disease but did not advance the product into formal clinical testing or toward approval. Dr. Jerry Vockley picked up the research with patients [ following ] compassionate use and then continued the clinical research of triheptanoin. In talking with Dr. Vockley, he indicated to me that he needed support in advancing the program. And so in 2013, we licensed exclusive worldwide rights from Baylor Research Institute to finish the development of triheptanoin, now called Dojolvi. Dr. Melanie Gillingham and Dr. Vockley, through their own clinical studies, have made significant contributions in the clinical development of Dojolvi for the full spectrum of patients with LC-FAOD. Their work, along with the work of many other investigators on the Dojolvi program has been published in peer-reviewed journals as the first-ever clinical studies of long-chain fatty acid oxidation diseases. The published Phase II study of Dojolvi conducted by Ultragenyx also demonstrates the effect of the therapy for patients with LC-FAOD, replicating the earlier results from many years of compassionate use. In addition to these investigators and other health care providers who were involved in the program, I would like to acknowledge the patients, their caregivers and their families who participated in clinical studies over many years through so many study visits, blood tests and other procedures. Today's approval is also a culmination of incredible collaboration across many people I mentioned, as well as the many different members of teams at Ultragenyx who have worked tirelessly to make this discovery a reality for patients with LC-FAOD. Based on all of the work that we and others have done, it's clear that many patients with LC-FAOD are not doing well on current management. LC-FAOD patients are affected early in life and it's a known cause of sudden infant death syndrome, also called SIDS, which led to the need for newborn screening for these disorders. Despite diagnosis in newborns and best available care, we've received approximately 100 compassionate use requests globally, many of which were emergency cases that have precipitously deteriorated in the ICU on heart-lung bypass. With the ever-increasing number of urgent requests that were often coming too late in patients' lives, we determined that we needed to seek approval with the currently data in hand to try to improve the care of these patients before the final crisis of their lives. Due to FDA's collaboration and willingness to evaluate the Phase II data available, and their effective use of the appropriate flexibility rare disease drug review, we now have an approved therapy available broadly to patients years earlier than had we waited and conducted a randomized Phase III trial. It's now our top priority to ensure that every patient in the U.S. with LC-FAOD who could benefit from Dojolvi gains access and receives treatment. Our goal with all of our medicines is that no patient goes without treatment in the U.S. due to financial reasons, and we continue this commitment with Dojolvi. I'll now turn the call over to Erik Harris to walk through the cost of therapy, our patient support programs, and our launch plans. Camille Bedrosian will then wrap up and discuss our ongoing work to advance the field's understanding of LC-FAOD and Dojolvi. We'll then turn to your questions. Erik, please proceed.

Thank you, Emil. Let me begin by sharing the commercial organization's excitement and enthusiasm following today's FDA approval. We strongly believe that cost should not be an impediment to access. It is our commitment to ensure affordable access for all patients diagnosed with LC-FAOD regardless of ability to pay. For patients who cannot pay, we commit to providing Dojolvi free of charge through our patient assistance program. We have taken great care to set a price for Dojolvi that will ensure a broad access for patients. Dojolvi will be available in 500-milliliter bottles and the wholesale acquisition cost per bottle is $4,875. This equates to an average net price of $138,000 per patient per year. However, this is not the price patients will actually pay. This net price includes discounts [ and assume ] compliance for patients on therapy and is based on the average daily caloric intake of patients. Because of the impact LC-FAOD can have on newborns, we want to ensure that patients receive treatment early in life. The resulting average net price for an infant is $46,000 for the first year of life. There were many factors that contributed to determining the price of Dojolvi, including the benefit to patients, the severity and life-threatening nature of the disease and the relatively small patient population with an estimated 2,000 to 3,500 patients in the U.S. We also took into account the lack of an FDA-approved treatment option while acknowledging the current use and cost of MCT oil. I will note that the price of Dojolvi is at the lower end of all therapies for inborn errors of metabolism that have been approved in the past 10 years and represents our commitment to responsible pricing. This pricing approach should result in broad access for patients and maximizes our ability to treat as many patients as possible. The vast majority of patients will have 0 or very low out-of-pocket costs for Dojolvi. We estimate that the majority of patients will be on commercial plans. These patients will have 0 out-of-pocket costs because their co-pays will be covered by our patient support plans. The remaining patients who are on government plans will have a small co-pay of $5 a month or less. I'll now turn to our launch plans and expectations. Our commercial infrastructure is already well established. We will leverage our expertise and relationships that we have built over the past 3 years, with Crysvita and Mepsevii to ensure that the Dojolvi launch is a success. In the early stages of the U.S. launch, we will focus on transitioning the approximately 80 patients currently on clinical drug in the U.S. to reimbursed commercial therapy. Our team has started conversations with physicians at our clinical sites to provide information about these transitions, which we expect to complete in the upcoming months, dependent upon payers' new-to-market coverage policies. The majority of patients with LC-FAOD are seen at approximately 160 specialist treatment centers. We know the health care providers at many of these centers through our efforts with Mepsevii and to some extent with Crysvita, as well as through our OTC and GSDIa clinical development programs, since the same doctors treat these diseases. With newborn screening in place for nearly 20 years, most LC-FAOD patients are already known, and there is a high awareness in the major genetic metabolic clinics. As a result, we will be able to launch with a relatively small and efficient team and plan to make fewer than 10 incremental hires on top of the team that we already have. Also want to acknowledge that we are launching this therapy during a challenging time with the COVID-19 pandemic. The work that we have done to adapt to the current situation for our other 2 approved therapies will be extremely helpful as we launch Dojolvi. The commercial team has found innovative ways to communicate with providers and patients to limit the impact on our commercial efforts. These adaptations, coupled with the ability -- patients' ability to take Dojolvi at home since it is an oral therapy, give us confidence that we will be able to have a successful launch. As we indicated before, we expect a gradual and steady build for Dojolvi in the initial quarters of launch. In some circumstances, the time to reimbursement could take up to 12 months, as payers establish new-to-market coverage policies. We expect the bulk of our 2020 revenue to continue to come from the EU Named Patient sales, with the U.S. gradually building over time. Having said this, we believe the value of Dojolvi to patients is high and that over time, the uptake will reflect the true value of the therapy. In the early quarters of the launch, we plan to provide some launch metrics to help characterize the strength and momentum of our launch. At this point, we are not providing any financial guidance, market assumptions or sales potential since it is difficult to predict how the various factors will impact initial uptake. We will continue to evaluate the possibility of providing guidance as we gain comfort through the ramp of the launch. I will now turn the call over to Camille.

Thank you, Erik. First and foremost, I also would like to express my gratitude to the patients, families, caregivers, dietitians and physicians who have been involved in the development of Dojolvi. The dedication and efforts of these individuals have brought us to this meaningful approval today. Emil spoke of our commitment to helping every patient who could benefit from our medicines get access. We accelerated our clinical development of Dojolvi as much as possible given the urgency to treat. We have provided Dojolvi to nearly 200 patients with LC-FAOD worldwide through expanded access, investigator-sponsored studies, and our clinical development program, with many patients on treatment now for over 10 years. The number of patients treated and their exposure duration represent one of the largest data sets collected for any rare disease drug ever. Now that Dojolvi is approved, our work does not stop. We will initiate a long-term disease monitoring program, or DMP, a fully sponsored program to collect high-quality data and support more learning and improvements in the care of LC-FAOD. This study will include at least 300 patients for an expected 10 years, with those on Dojolvi receiving commercial reimbursed therapy. Based on the responses to our ongoing DMP program for Crysvita, we expect patients, providers and payers also to respond favorably to this appropriate investment and the long-term outcomes of Dojolvi treatment. This program will be the single post-marketing clinical program and will include the post-marketing item that the FDA has required in one high-quality study. In addition to our progress in the United States, we will expand the reach of Dojolvi to patients in other regions of the world. We have submitted Dojolvi to Anvisa in Brazil and a potential marketing authorization in Brazil could follow this FDA approval. In Canada, we have been granted priority review, and we plan to file a new drug submission this year. We also continue our discussions with regulatory authorities in the EU. Named patient treatment in France and Italy have been growing steadily and will continue to grow while we seek agreement on our filing plan. With today's approval and the continued commitment of the entire community, there is a strong future with Dojolvi as the first approved therapy for patients with LC-FAOD. I will now turn the call back to Emil.

Thank you, Camille. Today's news marks the fourth U.S. FDA approval for Ultragenyx all in the last 3 years. We've provided medicines to treat a total of 9 diseases in our first 10 years as a company, now including the 6 separate enzyme deficiencies that make up LC-FAOD. We have a strong foundation with Crysvita and Mepsevii, and we continue to expand this with the FDA's recent approval of Crysvita for a second indication, the treatment of tumor-induced osteomalacia. Today's Dojolvi approval further establishes Ultragenyx as a major commercial [Audio Gap] company. Dojolvi has the potential to become a significant contributor to Ultragenyx's growth over time and will help fuel the future of the company. Beyond our approved products, our pipeline is very active with 2 gene therapeutic programs approaching Phase III studies later this year or early next. Our Angelman program recently initiated a Phase I/II and a diversified preclinical portfolio. We've come a long way in our first 10 years, and we look forward to continuing to grow as a company and help as many patients with rare diseases as we can. I'll now open up the call to questions. Operator, please provide the instructions for the Q&A.

[Operator Instructions] Our first question will come from Gena Wang with Barclays.

Congratulations on early approval with a very broad label. So my first question is regarding the pricing assumption. I mean you did mention the $138,000 for adults and $46,000 for newborns, that was after discount and a compliance. So what is your assumption of discount rate? And also, what is your assumption for compliance? My second question is regarding the 10 incremental hires. Would that cover 160 centers? Any synergies from existing sales?

Great. So 2 questions. I'll touch on price first and then let Erik fill in a little bit more. The $138,000 included an adjustment for gross to net as well as compliance. It was an average price for the patients. The $46,000 price was for the first year of life of patients who are on -- because the dosing is based on calories, they're actually on a higher dose per kilo, but they're for a very small size. So that's why there's that difference. So that's the pricing question. And I'll let -- Erik, do you want to add a little more on pricing? And then you can talk about the commercial investment required.

Yes. So just -- as far as the question, your question with regards to discounts and compliance. It's your typical discounts required for best government pricing. And then as far as compliance is concerned, it's consistent with what we saw in our clinical trials. As for...

Sorry. So would that be like government best pricing, that's 23%? And the normal compliance, we will consider like 15% to 20%, is that in line with your assumption?

Those are factors that -- those are factors based on a percent covered by government plans.

Yes. We're talking about growth at an...

And an average gross rent.

Gross rent.

Right.

We have given the bottle price, Erik, the total bottle price. I think people are trying to determine what the difference between the list prices and the combination may be, the gross to net. We haven't broken out the gross to net and compliance assumptions, but do you have that overall percent reduction?

I don't have that overall percent reduction just readily at hand.

Okay. Well, we can look for that. So tell us about the -- she wanted to know about the commercial leverage in the 160 centers.

Yes. So a very concentrated patient population, as we stated about 2,000 to 3,500 patients. I have about 160 of the metabolic genetic centers. And these are centers where we already have a strong presence and relationship, as I stated with regards following our efforts around Mepsevii, to some extent, Crysvita. And then our growing efforts with our clinical development program for OTC and GSDIa. Also, in many of these clinics, there's some awareness already of Dojolvi based on colleagues that have been involved in our clinical trials. As a result, we won't need many additional resources to be able to cover this physician population. So we're going to hire less than 10 incremental hires of field personnel, a combination of commercial field personnel and dietitians that will assist the patients and the providers with dosing and administration. And then we'll pretty much leverage our current infrastructure as it relates to our patient support services hub.

Our next question will come from Yaron Werber with Cowen.

This is Brendan on for Yaron. Congrats on the approval. I appreciate it, this is really helpful. I actually just really quickly wanted to ask, can you maybe just remind us and maybe give us a little sense of how you think the drug will be dosed over time? I know you mentioned it's dosed per kilo. I'm just kind of wondering if there's different things you look at in dosing pediatric versus adult patients. And then I actually just wanted to -- I'll have a follow-up about the pancreatic precautions.

It's actually dosed relative to calorie intake, to estimate about 30% of calorie intake is the target. So in a baby, the actual grams per kilo dosing is higher, it might be as high as 4 grams per kilo, whereas in adults it's closer to 1, so there's a range. And by the time you get to age, school-age children, you start reaching sort of a top dose level. And so the cost for a -- like a teenager and then an adult, are pretty much the same cost. There's kind of a flat-top distribution to it. So it's really based to target 30% of calorie intake in terms of the calories of drug.

Okay. All right. Great. And really quickly, just about the pancreatic precautions. Can you give us maybe just a sense of, if you actually even know, what percent of patients even have these low pancreatic enzymes or might be taking the lipase inhibitors? And if you think that's going to be any kind of a hurdle?

The pancreas thing is, I think, is purely theoretical since lipases come from the pancreas, but we have not seen anyone with a problem digesting the product related to the pancreas dysfunction. So it was purely theoretical risk, and I think the agency just wanted to call that out as one thing to watch for. But we haven't any evidence of patients who are unable to digest the product. And if you measure the digestion of the product, it's actually very fast in the GI tract.

Our next question will come from Laura Chico with Wedbush Securities.

I've just got two. I guess, one, I apologize if I missed it, but could you talk to perhaps the mix or breakdown in terms of the patient ages? Like what proportion of the market do you anticipate being the pediatric? And then second question, I thought that UX007 had a rare pediatric disease designation. I just wanted to confirm that the approval also mean receipt of another PRV. If so, I would be curious to hear more about your plans for that.

Yes. So the age distribution is complicated because there is a history of newborn screening in many states for some years, but there are also adult patients who may have not been -- who've been born before that. So that population is more uncertain. A large fraction of patients are going to be children and young adults. And there are fewer that older, mainly because they would have died in many cases already or they would not have been diagnosed. We haven't put out any more. I don't know if Erik, you wanted to add any more color on the distribution than that.

The only thing I would say, just in -- as we expect higher penetration among infants and children compared to adults in that patients had most risk of complications or more frequent events being put on therapy more than others.

Yes. Okay. On the PRV, we were not given the -- we were given standard approval rather than priority review. And therefore, would not be eligible for a PRV.

Our next question will come from Maury Raycroft with Jefferies.

Congrats on the update. I was wondering if you can remind what FDA requirements are for post-market data. Was there anything new that they specified upon approval? And what are your plans to complete those post-market requirements?

Well, one thing I'd state first is that we proposed this comprehensive disease monitoring program that Camille already described in the press release, which is our -- now has been accepted by FDA in 4 programs. And it's a novel approach to getting high-quality data, but also restricting the activity to one study, one clinical study, one good one, which is all [Audio Gap] all the patients are on commercial drug is the actually far more cost-effective solution. I'll let Camille talk about the one commitment request we've made, and that will be monitored in the disease monitoring program.

Thank you, Emil, and thanks for your question. Yes, we have 1 post-marketing requirement, and that is to follow patients who become pregnant while receiving Dojolvi. They will be followed prospectively. And then infants born will also be followed. And again, this single-arm follow-up will be conducted within the DMP.

Got it. That's helpful. And then just one follow-up question. If you could just remind what the doctor has to do to molecularly confirm LC-FAOD, and if you can provide any more specifics around that test -- the testing [ management ].

They would need to do sequencing to look at it. It's more difficult to do -- you can do a metabolic test with fiber [ OS ], but those are more difficult. The FDA is looking for sequencing, which is commonly done now, and we, of course, would support fully as part of our program that we do for all of our patients.

Our next question will come from Chris Raymond with Piper Sandler.

This is Nicole Gabreski on for Chris. Congrats on the early approval. I guess just one quick one. What can we expect just as far as launch metrics for Dojolvi moving forward? I guess, will this be similar to what we've seen for Crysvita?

I think Erik could answer that question, Erik?

Yes. So in the early quarters, we plan to provide some launch metrics to help characterize the strength and momentum of our launch. We haven't yet determined exactly what those metrics will be. I'll remind you that for Crysvita, we provided a number of prescribers, patient start forms and patients on reimbursed therapy.

We would expect it to be similar.

Yes, it will probably be similar. And the other thing I'd just point out is that we provided those for the first 6 quarters following the launch. And as we got more comfortable with the ramp, we moved to guidance.

Our next question will come from Cory Kasimov with JPMorgan.

This is Turner on for Cory. So understanding that the expectation is a bit of a slow launch due to new-to-market coverage policies, but given somewhat of the concentrated patient population at these centers at which you have a presence, are you seeing any pent-up demand whatsoever? And do you expect any initial bolus of patients at all?

Well, I'll start with the point that we did mention in the call today, which is that we've had 100 compassionate use requests, and majority of those from the U.S. So there's clearly pent-up demand to be getting those requests of compassionate use access for patients around. So there are some patients certainly out there, we expect. It's hard to know what that number will be and how it might affect the launch dynamic during the year, but we certainly would expect there'd be some doctors in that mode just because we have seen so much compassionate use. The other point is that Erik did put forth that there were 80 patients in trials who would be our first target. So I would look at that 80 and there'll probably be some others that we'll work on. I think the challenge is not that they're available and want treatment. The question is, how long does it take to get through the policies, process and the new-market approach that's taken by some insurers. So we think that will kind of provide some pacing to the early part of the launch. But we do think that with the controlled trial patients and some likely pent-up demand, we would expect the program should do well.

Our next question will come from Vincent Chen with Bernstein.

For the estimated 2,000 to 3,500 patients in the U.S., how does this break down between the various types of LC-FAOD, such as VLCAD, LCHAD, TFP? And if I think about the VLCAD population in particular, which is fairly large, but pretty variable in severity, what percentage of these do you include in the addressable markets? And then a quick follow-up, which is just how many VLCAD patients are diagnosed by newborn screening each year? And is it readily apparent at birth which of these patients are likely more or less severe?

Yes. So we haven't really broken down the severity of the 2,000, 3,500 because I don't think anyone quite has a firm grasp on exactly what the distribution is. I think you're right. I think people have looked at within certain states like California, there's been a publication on their [ cadence ] since VLCAD is clearly the most common. And then after that, you have the CPT, CPT II, LCHAD and TFP. The CACT version, CPT I is much lower. So VLCAD and a couple of others are probably the majority of the cases. And we believe there's around 100 diagnosed a year, something in that range, across the U.S. Now VLCAD does have heterogeneity. There are patients who could be in heart failure as infants, or patients that will have a type that may not require treatment. Obviously, patients that don't require any treatment, are extremely mild, we would not expect to be adopters of Dojolvi. But I think many of the physicians do have a sense of the types of severity that each mutation can provide. But the challenge generally in all genetic diseases, even if you know what you think a mutation is mild or moderate or within any individual family, within any particular genetic background, you can get a more severe result. And even within the same family, patients can vary with how severe they are. So I think most of the geneticists are very familiar with that variation, and they're going to look carefully at their patients. We do think that triheptanoin has shown benefit in VLCAD, young VLCAD patients, and particularly with the heart failure of [ one sign ], in some of the early individual compassionate use cases that we've seen. So we'd expect doctors to make a good judgment on what they know about their patients. And we think that the population, since it's getting newborn screening, will at least continue to expand as each year moves forward.

If you think about the -- just to clarify, if you think about the VLCAD population that's diagnosed on newborn screening, is it reasonable to think that many of these patients would be started on drug since, I guess, well, you may not wait to see whether they do really poorly before you start the drug? And in which case, they may continue. And I guess conceivably, now that you've got this combination of newborn screening and potentially a more effective drug, would you expect that population to grow over time?

Well, I think you -- one of the things that's very tricky is that you don't really know how the patient is going to do. Because FAOD is an episodic disease. It can be going along but are really right on the edge, and all they need is one cold as a 3-month old to send them into a crisis coma. And in fact, before newborn screening, 38% of the deaths were happening essentially in the first year of life because of a single event. So there is a high sense of risk and urgency in being preventive of potential issues coming up. So we would expect doctors would have to consider all of these issues and what's been seen with the product to make decisions on how to manage patients. But we do believe that if triheptanoin is a better option in the beginning, that the patients once on it, would likely stay on it. I think demonstrating that it is a better first-line will be one of the things we are going to be looking at in our disease monitoring program and using other real-world evidence to look at young patients started on therapy and the risk going forward. The kind of thing we'd like to collect more data on, based on our experience, is treating infants. We have quite a few infants who are in compassionate use serious situation, we think that triheptanoin does or Dojolvi has a role in treating those patients. And whether it gets used prophylactically by a doctor, I think, will depend on their own personal experiences and their belief on the need to get ahead of a problem like that rather than react to it after it happens.

And our next question will come from Jeff Hung with Morgan Stanley.

Congratulations on the approval. Erik, did I hear correctly that most LC-FAOD patients are already known? And if that's the case, can you talk about what factors are involved in getting from identifying the patients to treating them since it sounds like the initial focus is on transitioning the 80 patients to commercial therapy? I'm just trying to get a sense for any limiting factors for the number of patients you're able to treat. Thanks.

As far as your question [ has gone ], I mean, the first and foremost is just to -- [ we've first got awareness that they're ] launched, that the product is now available. And then, what we do know from our discussions with payers is that while the indication is broad, highly likely that the payers will require this prior authorization confirmation of diagnosis -- of being diagnosed with LC-FAOD either molecularly or genetically. And as Emil stated, one of the things we're going to do is provide, support that at no cost to the patients. So the initial step is getting these patients into the clinic, get them confirmed -- from past experience, we know that can take some time to get confirmation. And then they will then go through the reimbursement process. So -- which can take several months to get them on to commercial reimbursed product. In the meantime, we will support those patients with regards to keeping them on therapy through our bridge or fast start programs.

Yes. I think one thing I'd add is, I'm sure that there are patients who are having more problems, that will be more driven to adopt more rapidly than those that may not. But the fact that the label is broad gives doctors an opportunity to treat a wide array of patient severity, and we think that's really important in how the product does over the long haul.

So can I just confirm again, though, that I heard correctly that most LC-FAOD patients are already known? Or is there any additional work that needs to be done on identifying additional patients?

Most of the patients are known to the system because they've been through the newborn screening process. Patients that are over the age of 20 or so, may be more -- may have more patients who are having muscle problems and don't know what it's about, haven't figured it out. So it's possible above the older age range that there are people who have survived and have recurring problems that are not been properly figured out. I recently saw a video of just a case like that, it was on a TV show. And on the TV show, they made the diagnosis of LC-FAOD, which was exciting for us to see. So there are some patients who are older, where they were born before newborn screening applied in their state. So those are the things that are unknown. We will have to continue to work to find those patients. But the difficulty is they won't be necessarily at the centers because they would not have -- they wouldn't know they should go to a metabolic genetic center. They might be going to a muscle doctor or other things. But we've been very good at using other tools to find patients that might be highly likely. We've been doing that for XLH using other data sets and AI-type technology to help identify possible patients and looking for additional patients of those older age groups. But as Erik said earlier, I think the young patients tend to have the most severe problems. And so we'd expect that most of the patients should be known and diagnosed.

I'm showing no further questions in the queue at this time. I would now like to turn the call back over to Emil for any further remarks.

Thank you, and thanks, everyone, for joining us today. Obviously, an important day getting Dojolvi approved, our fourth product as a company in our 10 years of existence. An important disease area, long-chain fatty acid diseases have never been addressed by any drug or any company, and we're very proud to have been the first company that moved a drug forward for a disease this severe and be able to provide a new option for their treatment. We do thank, again, all the patients, investigators who've done the work in helping us as well as the Ultragenyx team. We've done a spectacular job working through this product the last few years to deliver today to patients in the U.S. and hopefully, patients elsewhere around the world soon. Thank you for joining us today. Goodbye.

Ladies and gentlemen, this concludes today's conference call. Thank you for your participation. You may now disconnect.