Click to expand.
Transcript highlights via SeekingAlpha.
Prepared Remarks
Note EU name of LOJUXTA vs JUXTAPID in USA.
27-0 vote among member countries
"With our estimate of approximately 3,000 adult HoFH patients, the EU is a critical market for Aegerion, and the CHMP positive opinion is an important milestone for patients with this disease. We anticipate the European Commission will ratify the positive opinion to grant marketing authorization of LOJUXTA in all 27 union-member countries in the third quarter this year. Upon that approval, we will begin our launch on a country-by-country basis.
As many of you are aware, we can begin selling in countries such as Germany and the U.K. upon approval. Whereas in countries such as France, it may take up to 9 months, or even longer, to obtain approval for reimbursement. For that reason, we've been cautious to guide you that we do not expect material revenue from Europe in 2013. Revenue contribution from the EU will begin in a meaningful manner beginning in 2014 and will accelerate from there.
We have filed for approval of lomitapide in Brazil, which is critically important to our long-range plan as we anticipate Brazil will be 1 of the top 3 markets. We also met with the Mexican regulatory authorities recently and came away from the meeting with clear guidance on our path to commercialization in that country. We received exceptions from a -- exemptions, excuse me, we received exemptions from a number of requirements typically expected of new molecules, and we're optimistic that the registration process will be rapid. We expect Mexico will also be a significant market for lomitapide.
In Canada, our filing has been accepted and is under review. There's a founders effect in the French-Canadian population, and we have a meaningful number of patients already identified in Canada. Finally, as you're aware, the Japanese market is an important one. Following the successful completion of the Japanese PK/PD study expected in the fourth quarter, we plan to have discussions with the Japanese regulatory authorities and a contract with a CRO at that time to conduct a small therapeutic study in Japanese HoFH patients. If we receive no further request for additional data or analysis, we plan to dose the first patient in this therapeutic study in either the fourth quarter of 2013 or in the first quarter of 2014. Assuming we are able to file, following completion of the 26-week efficacy phase, we plan to be in a position to submit our Japanese NDA as early as the first half of 2015."
Q&A Session:
Just curious, in the EU, which countries currently use genetic confirmation?
let me emphasize that genetic confirmation and testing is not required; it is recommended. And we believe this gives the physician, in all 27 member countries, the ability to use their judgment on how to diagnose these patients. Genetic testing in Europe is done slightly more frequently than the U.S., but it is still rare. It is not done broadly in either area, or anywhere in the world, frankly. So we feel comfortable that, again, the EMEA, they got it right. They understand the risk benefit of this product, and they're allowing the physician to make a diagnosis-judgment call, and they're recommending, if possible, to do genetic testing, but not required. So it's not required to be very specific. About your question, you had asked, where is it required and what countries now? There's no requirement in any country now right now, in Turkey, we can commercialize on an named-patient basis and ask for approval patient by patient, like we're doing in Brazil. So we have scripted patients in Turkey today. We have over 100 patients that have been identified in Turkey. And we've gotten a good -- we've received good traction early on with scripts in Turkey. So we can sell again, at a U.S. price, named-patient basis in Turkey today.
what kind of delay you expect between -- in Europe, between patient getting a prescription and then patient actually starting on therapy? I think it is roughly 3 to 4 months in the U.S., so do you expect that -- a similar difference between patients starting on therapy in Europe as well?
It is one of -- it's going to depend country by country. And in certain countries, we will need to wait for reimbursement, and we'll do that and we'll work through that process.
And it will be relatively the same timing as the U.S. or slower. That's one element that will slow down the revenue growth in Europe. The other thing I would caution you about in Europe is that the European revenue business, in our field, the last 5 years, has slowed. And I think companies have gotten out over their skis in expectations, and we're just guiding you to be cautious about the revenue growth in Europe. It will not be at the pace of the U.S. It's going to be a critically important market to us. We see it being the same size roughly as the U.S. market, but it will build slower than the U.S. growth
Marc, with respect to the 4,000 to 5,000 patients on therapy that you envision down the road, what portion of that would you estimate could be U.S., Europe and then outside of both of those regions?
CEO wouldn't answer. all three "meaningful" down the road 5-7 yrs
the first part of that question I got which is, is the 3,000 is taking into consideration the countries that in the next 5 years we're probably not going to go into with any significant firepower? It doesn't look at those countries. I try represent the number in an accurate way of what we're going to market to and where we plan on going. So in the countries that we plan on going commercially into, there are some countries in the 27-member countries that they get very challenged in reimbursement, if at all, and the size of those markets are markets that we may, in the future, set up a local distributor until it becomes sizable enough where we'd replace the local distributor with, with one of our own employees. But some of those countries, we don't plan on being in, in a meaningful way in the next 5 years. And those countries are not included in that number.
First one is, do you still -- are you still standing by the fact that if you do not get the pricing that you want in European countries, that you will not sell in those countries?
Yes, Kim, that's very important in our strategy on global basis. If you give on price in any area of the world, it's a slippery slope. Precedent [ph] pricing, I think, is appropriate. I'm a complete supporter of precedent pricing. I don't know why we should feel comfortable pricing a product 40% less in a certain market. We should -- so we will hold price. If it takes a little bit longer in certain countries, we will wait it out and we'll hold price on a global basis.
Salesforce
we haven't made a public on the number of sales reps that we will launch within Europe. We're looking at it country by country. And I can tell you this, that they'll be tranched just like a tranche expenditure here.
Prepared Remarks
Note EU name of LOJUXTA vs JUXTAPID in USA.
27-0 vote among member countries
"With our estimate of approximately 3,000 adult HoFH patients, the EU is a critical market for Aegerion, and the CHMP positive opinion is an important milestone for patients with this disease. We anticipate the European Commission will ratify the positive opinion to grant marketing authorization of LOJUXTA in all 27 union-member countries in the third quarter this year. Upon that approval, we will begin our launch on a country-by-country basis.
As many of you are aware, we can begin selling in countries such as Germany and the U.K. upon approval. Whereas in countries such as France, it may take up to 9 months, or even longer, to obtain approval for reimbursement. For that reason, we've been cautious to guide you that we do not expect material revenue from Europe in 2013. Revenue contribution from the EU will begin in a meaningful manner beginning in 2014 and will accelerate from there.
We have filed for approval of lomitapide in Brazil, which is critically important to our long-range plan as we anticipate Brazil will be 1 of the top 3 markets. We also met with the Mexican regulatory authorities recently and came away from the meeting with clear guidance on our path to commercialization in that country. We received exceptions from a -- exemptions, excuse me, we received exemptions from a number of requirements typically expected of new molecules, and we're optimistic that the registration process will be rapid. We expect Mexico will also be a significant market for lomitapide.
In Canada, our filing has been accepted and is under review. There's a founders effect in the French-Canadian population, and we have a meaningful number of patients already identified in Canada. Finally, as you're aware, the Japanese market is an important one. Following the successful completion of the Japanese PK/PD study expected in the fourth quarter, we plan to have discussions with the Japanese regulatory authorities and a contract with a CRO at that time to conduct a small therapeutic study in Japanese HoFH patients. If we receive no further request for additional data or analysis, we plan to dose the first patient in this therapeutic study in either the fourth quarter of 2013 or in the first quarter of 2014. Assuming we are able to file, following completion of the 26-week efficacy phase, we plan to be in a position to submit our Japanese NDA as early as the first half of 2015."
Q&A Session:
Just curious, in the EU, which countries currently use genetic confirmation?
let me emphasize that genetic confirmation and testing is not required; it is recommended. And we believe this gives the physician, in all 27 member countries, the ability to use their judgment on how to diagnose these patients. Genetic testing in Europe is done slightly more frequently than the U.S., but it is still rare. It is not done broadly in either area, or anywhere in the world, frankly. So we feel comfortable that, again, the EMEA, they got it right. They understand the risk benefit of this product, and they're allowing the physician to make a diagnosis-judgment call, and they're recommending, if possible, to do genetic testing, but not required. So it's not required to be very specific. About your question, you had asked, where is it required and what countries now? There's no requirement in any country now right now, in Turkey, we can commercialize on an named-patient basis and ask for approval patient by patient, like we're doing in Brazil. So we have scripted patients in Turkey today. We have over 100 patients that have been identified in Turkey. And we've gotten a good -- we've received good traction early on with scripts in Turkey. So we can sell again, at a U.S. price, named-patient basis in Turkey today.
what kind of delay you expect between -- in Europe, between patient getting a prescription and then patient actually starting on therapy? I think it is roughly 3 to 4 months in the U.S., so do you expect that -- a similar difference between patients starting on therapy in Europe as well?
It is one of -- it's going to depend country by country. And in certain countries, we will need to wait for reimbursement, and we'll do that and we'll work through that process.
And it will be relatively the same timing as the U.S. or slower. That's one element that will slow down the revenue growth in Europe. The other thing I would caution you about in Europe is that the European revenue business, in our field, the last 5 years, has slowed. And I think companies have gotten out over their skis in expectations, and we're just guiding you to be cautious about the revenue growth in Europe. It will not be at the pace of the U.S. It's going to be a critically important market to us. We see it being the same size roughly as the U.S. market, but it will build slower than the U.S. growth
Marc, with respect to the 4,000 to 5,000 patients on therapy that you envision down the road, what portion of that would you estimate could be U.S., Europe and then outside of both of those regions?
CEO wouldn't answer. all three "meaningful" down the road 5-7 yrs
the first part of that question I got which is, is the 3,000 is taking into consideration the countries that in the next 5 years we're probably not going to go into with any significant firepower? It doesn't look at those countries. I try represent the number in an accurate way of what we're going to market to and where we plan on going. So in the countries that we plan on going commercially into, there are some countries in the 27-member countries that they get very challenged in reimbursement, if at all, and the size of those markets are markets that we may, in the future, set up a local distributor until it becomes sizable enough where we'd replace the local distributor with, with one of our own employees. But some of those countries, we don't plan on being in, in a meaningful way in the next 5 years. And those countries are not included in that number.
First one is, do you still -- are you still standing by the fact that if you do not get the pricing that you want in European countries, that you will not sell in those countries?
Yes, Kim, that's very important in our strategy on global basis. If you give on price in any area of the world, it's a slippery slope. Precedent [ph] pricing, I think, is appropriate. I'm a complete supporter of precedent pricing. I don't know why we should feel comfortable pricing a product 40% less in a certain market. We should -- so we will hold price. If it takes a little bit longer in certain countries, we will wait it out and we'll hold price on a global basis.
Salesforce
we haven't made a public on the number of sales reps that we will launch within Europe. We're looking at it country by country. And I can tell you this, that they'll be tranched just like a tranche expenditure here.
RSS Feed
