CAMBRIDGE, Mass. (
) -- Following U.S. approval in December,
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is now in the early days of the commercial launch of Juxtapid for the treatment of homozygous familial hypercholesterolemia (HoFH), a rare genetic disease that prevents cholesterol from being cleared from the body. As a result, HoFH patients suffer from the buildup of fatty deposit in their tissues and blood and are at severe risk for fatal heart attacks.
On Tuesday, I spoke with Aegerion CEO Marc Beer for an update on the Juxtapid launch and future plans for the company. We also discussed orphan drug pricing and insurance reimbursement -- an issue of increasing focus and concern for drug companies and investors.
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, a competitor, also came up in our conversation, which took place hours before FDA approved Isis' HoFH drug Kynamro.
One year of Juxtapid therapy costs $250,000, on a blended basis. Aegerion expects to have 250-300 HoFH patients on drug by the end of 2013, with revenue in the range of $15-25 million. The company has 1,500 HoFH patients in the U.S. in its Juxtapid patient tracker database and believes there may be as many as 3,000 HoFH patients in the U.S.
I began the conversation with Beer asking about the steps Aegerion is taking to get Juxtapid's launch off to a strong start.
First, we're focused on training physicians in our REMS [risk management] program. We're watching the number of physicians trained closely and we're right on track where we want to be. In the first two or three days, 50 physicians asked for training, which means they have patients who can benefit from Juxtapid. We have to make sure that doctors know how to put the right patients on this drug.
Second, we're working on moving patients through the pre-authorization phase. We have forecasted that it will take 4-5 months to do that, on average. We're watching the first quarter closely to see how long it will take to get patients through the insurance coverage process.
Lastly, we're looking at the patients who have prescriptions, looking at the severity of the patients in order to forecast the clinical utility of Juxtapid when it's in the hands of treaters versus patients in our patient tracker database.
Describe the feedback you're getting from insurance companies regarding Juxtapid reimbursement.
Juxtapid is a true orphan drug in that the morbidity and mortality of HoFH is severe and our drug is disease modifying, so we always believed that insurers would respect our price point and reimburse...
But we had to price the product right and communicate with insurers. This is work that we did in the last six months of 2012. The early indications are very positive. We visited 85% of the U.S. population's insurance carriers prior to the launch and we continue to have those meetings. In the early days of processing pre-authorizations, we are getting no sense of pushback or restrictions from insurance companies. If we were, we'd be seeing demands for patients to try certain medicines first or to undergo apheresis, but we haven't seen anything like that yet.
Many people define orphan and ultra orphan drugs just by the small number of patients diagnosed with the disease, but you believe the drug's ability to modify the disease needs to be taken into account. Is this being driven by FDA or the insurance companies who must ultimately pay for these drugs?
It's coming from the insurance companies... If you have a marginal drug, even if it's in an orphan disease, insurance companies are pushing back.
Note: I interviewed Beer on Tuesday afternoon so FDA hadn't yet announced the FDA approval of Kynamro, a competing HoFH drug from Isis Pharmaceuticals that will be marketed by the Genzyme unit of
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Care to make a prediction about whether or not FDA will approve Kynamro?
I expecting a press release any hour that it [Kynamro] is approved. I always thought that both products would be approved. Both are needed but Juxtapid is much more commercially viable. I was surprised Kynamro was not approved in Europe.
I believe Juxtapid will be the first line treatment for HoFH. These patients are in dire straits, they live in fear of a cardiovascular event, but we are not going to get every patient to goal so to have another tool for patients and physicians is important.
Sanofi hasn't announced Kynamro pricing yet but consensus seems to be that it will be priced at a significant discount to Juxtapid. Does this worry you? Will a cheaper competitor change your strategy or affect the Juxtapid launch?