Table of Contents
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`U.S. SECURITIES AND EXCHANGE COMMISSION
`WASHINGTON, DC 20549
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`FORM 10-K
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`(Mark One)
` ANNUAL REPORT PURSUANT TO SECTION 13 OR 15 (d) OF THE SECURITIES EXCHANGE ACT OF 1934
`For the fiscal year ended December 31, 2014
` TRANSITION REPORT PURSUANT TO SECTION 13 OR 15 (d) OF THE SECURITIES EXCHANGE ACT OF 1934
`For the transition period from to
`Commission file Number: 001-34921
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`AEGERION PHARMACEUTICALS, INC.
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`(Exact Name of Registrant as Specified in Its Charter)
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`20-2960116
`Delaware
`(IRS Employer
`(State or Other Jurisdiction of
`Incorporation or Organization)
`Identification Number)
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`One Main Street, Suite 800, Cambridge, Massachusetts 02142
`(Address of Principal Executive Offices, including Zip Code)
`617-500-7867
`(Registrant’s telephone number, including area code)
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`SECURITIES REGISTERED PURSUANT TO SECTION 12(b) OF THE ACT:
`Common Stock, $0.001 Par Value
`The NASDAQ Global Select Market
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`SECURITIES REGISTERED PURSUANT TO SECTION 12(g) OF THE ACT:
`None
`(Title of Class)
`
`Indicate by check mark if the registrant is a well-known seasoned issuer, as defined in Rule 405 of the Securities Act. Yes  No 
`Indicate by check mark if the registrant is not required to file reports pursuant to Section 13 or 15(d) of the Act. Yes  No 
`Indicate by check mark whether the registrant: (1) has filed all reports required to be filed by Section 13 or 15(d) of the Securities Exchange
`Act of 1934 during the preceding 12 months (or for such shorter period that the registrant was required to file such reports), and (2) has been
`subject to such filing requirements for the past 90 days. Yes  No 
`Indicate by check mark whether the registrant has submitted electronically and posted on its corporate Website, if any, every Interactive
`Data File required to be submitted and posted pursuant to Rule 405 of Regulation S-T during the preceding 12 months (or for such shorter period
`that the registrant was required to submit and post such files). Yes  No 
`Indicate by check mark if disclosure of delinquent filers pursuant to Item 405 of Regulation S-K is not contained herein, and will not be
`contained, to the best of registrant’s knowledge, in definitive proxy or information statements incorporated by reference in Part III of this
`Form 10-K or any amendment to this Form 10-K. 
`Indicate by check mark whether the registrant is a large accelerated filer, an accelerated filer, a non-accelerated filer, or a smaller reporting
`company. See definitions of “large accelerated filer,” “accelerated filer,” and “smaller reporting company” in Rule 12b-2 of the Exchange Act.
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`Large accelerated filer 
` Accelerated filer
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`Non-accelerated filer  (Do not check if a smaller reporting company)
` Smaller reporting company 
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`Indicate by check mark whether the registrant is a shell company (as defined in Rule 12b-2 of the Exchange Act). Yes  No 
`The aggregate market value of the voting and non-voting common equity held by non-affiliates of the registrant as of June 30, 2014 was
`approximately $915,210,330, based upon the closing price on the NASDAQ Global Market reported for such date.
`As of February 17, 2015, 28,539,381 shares of the registrant’s common stock were outstanding.
`DOCUMENTS INCORPORATED BY REFERENCE
`Portions of the registrant’s definitive Proxy Statement for its 2015 Annual Meeting of Shareholders are incorporated by reference into
`Part III of this Annual Report on Form 10-K
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`Table of Contents
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`FORM 10-K
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`TABLE OF CONTENTS
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`PART I
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` Business
` Risk Factors
` Unresolved Staff Comments
` Properties
` Legal Proceedings
` Mine Safety Disclosures
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`PART II
` Market for Registrant’s Common Equity, Related Shareholder Matters and Issuer Purchases of Equity Securities
` Selected Financial Data
` Management’s Discussion and Analysis of Financial Condition and Results of Operations
` Quantitative and Qualitative Disclosures About Market Risk
` Consolidated Financial Statements and Supplementary Data
` Changes in and Disagreements With Accountants on Accounting and Financial Disclosure
` Controls and Procedures
` Other Information
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`PART III
` Directors, Executive Officers and Corporate Governance
` Executive Compensation
` Security Ownership of Certain Beneficial Owners and Management and Related Shareholder Matters
` Certain Relationships and Related Transactions, and Director Independence
` Principal Accounting Fees and Services
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`Item 1.
`Item 1A.
`Item 1B.
`Item 2.
`Item 3.
`Item 4.
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`Item 5.
`Item 6.
`Item 7.
`Item 7A.
`Item 8.
`Item 9.
`Item 9A.
`Item 9B.
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`Item 10.
`Item 11.
`Item 12.
`Item 13.
`Item 14.
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`Item 15.
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` Exhibits and Financial Statement Schedules
`SIGNATURES
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`PART IV
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`Forward-Looking Statements
`All statements included or incorporated by reference into this Annual Report on Form 10-K, or Annual Report, other than statements or
`characterizations of historical fact, are forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995.
`Forward-looking statements are often identified by words such as “anticipates,” “expects,” “intends,” “plans,” “predicts,” “believes,” “seeks,”
`“estimates,” “forecasts,” “may,” “will,” “should,” “would,” “could,” “potential,” “continue,” “ongoing” and similar expressions, and variations
`or negatives of these words. Examples of forward-looking statements contained in this Annual Report include our statements regarding: the
`commercial potential for our products; our estimates as to the potential number of patients with the diseases for which our products are
`approved; our expectations with respect to reimbursement of our products in the United States; our expectations with respect to pricing and
`reimbursement approvals required for lomitapide in countries of the European Union, Mexico, Canada, and other countries in which we receive,
`or have received, marketing approval for lomitapide; our expectations with respect to named patient sales of our products in Brazil and in other
`countries where such sales are permitted; the potential for and possible timing of approval of our products in countries where we have not yet
`obtained approval; plans for further clinical development of our products; our expectations regarding possible future filings for approval of
`lomitapide in Japan and for approval of metreleptin in the European Union; our plans for commercial marketing, sales, manufacturing and
`distribution of our products; our expectations with respect to the impact of competition on our future operations and results; our beliefs with
`respect to our intellectual property portfolio for our products and the extent to which it protects us; our expectations regarding the availability of
`data and marketing exclusivity in the United States, the European Union and other countries; our view of ongoing government investigations and
`stockholder litigation and the possible impact of each on our business; our forecasts regarding sales of our products, our future expenses, our
`cash position and the timing of any future need for additional capital to fund operations; and our plans to acquire rights to one or more product
`candidates.
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`The forward-looking statements contained in this Annual Report and in the documents incorporated into this Annual Report by reference
`are based on our current beliefs and assumptions with respect to future events, all of which are subject to change. Forward-looking statements
`are not guarantees of future performance, and are subject to risks, uncertainties and assumptions that are difficult to predict, including those
`discussed in “Risk Factors” in Part I, Item 1A of this Annual Report. It is not possible for us to predict all risks, nor can we assess the impact of
`all factors on our business or the extent to which any factor, or combination of factors may impact our operations or results. New risks may
`emerge from time to time. Past financial or operating performance is not necessarily a reliable indicator of future performance. Given these risks
`and uncertainties, we can give no assurances that any of the events anticipated by the forward-looking statements will occur or, if any of them
`does occur or, alternatively, if any of the events described as a risk were to occur, what impact such event will have on our results of operations
`and financial condition. Our actual results could differ materially and adversely from those expressed in any forward-looking statement in this
`Annual Report or in our other filings with the Securities and Exchange Commission.
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`Except as required by law, we undertake no obligation to revise our forward-looking statements to reflect events or circumstances that arise
`after the date of this Annual Report or the respective dates of documents incorporated into this Annual Report by reference that include forward-
`looking statements. Thus, you should not assume that our silence over time means that actual events are bearing out as expressed or implied in
`these forward-looking statements.
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`In this Annual Report, “Aegerion Pharmaceuticals, Inc.,” “Aegerion,” the “Company,” “we,” “us” and “our” refer to Aegerion
`Pharmaceuticals, Inc. taken as a whole, unless otherwise noted.
`
`Trademarks
`Aegerion, JUXTAPID, LOJUXTA and MYALEPT are trademarks of Aegerion. All other trademarks referenced in this Form 10-K are the
`property of their respective owners.
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`Business.
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`Item 1.
`Overview
`We are a biopharmaceutical company dedicated to the development and commercialization of innovative therapies for patients with
`debilitating rare diseases.
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`PART I
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`Our first product, lomitapide, received marketing approval, under the brand name JUXTAPID (lomitapide) capsules (“JUXTAPID”),
`®
`from the U.S. Food and Drug Administration (“FDA”) in late December 2012, as an adjunct to a low-fat diet and other lipid-lowering treatments,
`including low-density lipoprotein (“LDL”) apheresis where available, to reduce low-density lipoprotein cholesterol (“LDL-C”), total cholesterol
`(“TC”), apolipoprotein B (“apo B”) and non-high-density lipoprotein cholesterol (“non-HDL-C”) in adult patients with homozygous familial
`hypercholesterolemia (“HoFH”). We launched JUXTAPID in the U.S. in late January 2013. In July 2013, we received marketing authorization
`for lomitapide in the European Union (“EU”), under the brand name LOJUXTA (lomitapide) hard capsules (“LOJUXTA”), as a treatment for
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`HoFH in adults. Lomitapide is also approved for the treatment of HoFH in Mexico, Canada, and a small number of other countries. We sell
`lomitapide, on a named patient basis, in Brazil and in a limited number of other countries outside the U.S. where a mechanism exists based on
`the U.S. or the EU approval.
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`We acquired our second product, metreleptin, in January 2015, pursuant to an asset purchase agreement (the “Asset Purchase Agreement”)
`dated November 5, 2014 with Amylin Pharmaceuticals, LLC (“Amylin”) and AstraZeneca Pharmaceuticals LP, an affiliate of Amylin (together
`referred to as “AstraZeneca”). Metreleptin, a recombinant analog of human leptin, is currently marketed in the U.S. under the brand name
`MYALEPT (metreleptin) for injection (“MYALEPT”). MYALEPT received marketing approval from the FDA in February 2014 as an adjunct
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`to diet as replacement therapy to treat the complications of leptin deficiency in patients with congenital or acquired generalized lipodystrophy
`(“GL”). Under the terms of the Asset Purchase Agreement, we paid AstraZeneca $325.0 million to acquire the global rights to develop,
`manufacture and commercialize metreleptin, subject to an existing distributor license with Shionogi & Co., Ltd. (“Shionogi”) covering Japan,
`South Korea and Taiwan. The distribution agreement with Shionogi was assigned to us as part of the transaction. We also assumed certain other
`assets and liabilities of AstraZeneca related to the metreleptin program.
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`We expect that our near-term efforts will be focused on:
` maintaining market acceptance of JUXTAPID as a treatment for adult HoFH patients in the U.S., particularly in light of the
`•
`anticipated introduction of competitive products, and continuing to support named patient sales of lomitapide as a treatment for
`HoFH in Brazil and in other key countries where such sales are permitted;
` building and maintaining market acceptance for MYALEPT in the U.S. for the treatment of complications of leptin deficiency in GL
`patients, and initiating named patient sales of metreleptin in GL in Brazil and other key countries where such sales are permitted as a
`result of the U.S. approval;
` gaining pricing and reimbursement approvals for lomitapide in key EU markets, Mexico, and Canada;
` gaining regulatory and pricing and reimbursement approvals to market our products in key countries in which the products are not
`currently approved, including filing a Marketing Authorization Application (“MAA”) with the European Medicines Agency
`(“EMA”) seeking marketing approval of metreleptin in the EU as a treatment for complications of leptin deficiency in GL patients,
`and, if approved, commencing commercialization efforts in those markets where it makes commercial sense to do so;
` minimizing the number of patients who are eligible to receive but decide not to commence treatment with our products or who
`discontinue treatment, including with lomitapide, due to tolerability issues and with metreleptin, due to its route of administration, as
`an injection, through activities such as patient support programs, to the extent permitted in a particular country;
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` clinical development activities to support a potential marketing authorization application for lomitapide in HoFH in Japan, and in
`support of our planned clinical study of lomitapide in pediatric HoFH patients;
` evaluating the potential for future clinical development of metreleptin in additional indications; and
` assessment, and possible acquisition, of potential new product opportunities targeted at rare diseases where we believe we can
`leverage our infrastructure and expertise.
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`Prior to 2015, we generated revenues solely from sales of lomitapide. In the near-term, we expect that the majority of our revenues will
`continue to be derived from sales of our products in the U.S. We also expect to generate revenues from sales of lomitapide in those countries
`outside the U.S. in which we have or receive marketing approval, and are able to obtain pricing and reimbursement approval at acceptable levels,
`and from sales of both our products in a limited number of other countries where they are, or may in the future be, available on a named patient
`sale basis as a result of existing approvals. We expect that named patient sales of lomitapide in Brazil in the near term will continue to be our
`second largest source of revenues for lomitapide, on a country-by-country basis. We expect to begin generating revenues from named patient
`sales of metreleptin in Brazil based on U.S. approval in the second half of 2015. We expect net product sales from named patient sales to
`fluctuate quarter-over-quarter significantly more than sales in the U.S. In some countries, including Brazil, orders for named patient sales are for
`multiple months of therapy which can lead to an unevenness in orders. In addition, net product sales from named patient sales may fluctuate
`quarter-over-quarter as a result of government actions, economic pressures and political unrest. For example, with respect to named patient sales
`of lomitapide in Brazil in 2014, we experienced longer than expected turn-around times between price quotation and order at the federal level,
`and delays in receipt of orders from the state government of São Paolo as a result of an ongoing São Paolo investigation focused on determining
`whether there has been any violation of Brazilian anti-corruption laws in connection with prescriptions written for lomitapide in São Paolo. A
`similar investigation has also been initiated by the federal government in Brazil.
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`We have submitted documentation seeking pricing and reimbursement approvals for lomitapide from governmental authorities in key
`markets of the EU, and are seeking such approvals from governmental authorities, social funds and private payers in Mexico and Canada. We
`anticipate reimbursement decisions in some of those countries in 2015. In March 2014, the reimbursement authority in Germany, the G-BA
`(Gemeinsamer Bundesausschuss) deemed our dossier for LOJUXTA to be incomplete as a result of certain technical deficiencies. As a result of
`the technical deficiencies, LOJUXTA was automatically put into the category of “no additional benefit” under the G-BA process, without a
`review of the clinical merits, which limits the reimbursement level significantly. After the G-BA assessment, we withdrew LOJUXTA from the
`German market in July 2014. We intend to re-file our dossier for LOJUXTA in June 2015, which we expect would result in an assessment by the
`G-BA in late 2015.
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`During the year ended December 31, 2014, we generated approximately $158.4 million of revenues from net product sales of lomitapide,
`of which $143.4 million was derived from prescriptions for lomitapide written in the U.S., and $15.0 million was derived from prescriptions for
`lomitapide written outside the U.S., primarily in Brazil. We did not generate revenues from sales of metreleptin in the year ended December 31,
`2014, as we had not yet completed acquisition of the product from AstraZeneca. As of December 31, 2014, we had approximately $375.9
`million in cash, cash equivalents and marketable securities on hand, of which $325.0 million was used to acquire MYALEPT in January 2015.
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`HoFH
`HoFH is a serious, rare genetic disease that impairs the function of the receptor responsible for removing LDL-C (“bad” cholesterol) from
`the blood. An impairment of low density lipoprotein receptor (“LDL-R”) function results in significant elevation of blood cholesterol levels.
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`Cholesterol is a naturally occurring molecule that is transported in the blood. The liver and the intestines are the two main sites where
`cholesterol is packaged and released within the body. The liver synthesizes cholesterol, and provides the body’s intrinsic supply. The intestines
`are the conduit through which cholesterol enters the body for metabolism. The delivery of cholesterol to peripheral cells in the body provides
`necessary sources of cellular energy and cell structure. However, excess levels of cholesterol in the blood, also known as hypercholesterolemia,
`can be the source of significant diseases in humans.
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`HoFH is most commonly caused by genetic mutations in both alleles of the LDL-R gene, but can also be caused by mutations in other
`genes. To date, more than 1,700 mutations have been identified that can impair the function of the LDL-R, with some mutations leading to a
`total lack of LDL-R activity and others leading to significantly reduced activity in LDL-R. As a result of elevated levels of LDL-C, HoFH
`patients often develop premature and progressive atherosclerosis, a narrowing or blocking of the arteries, and are at very high risk of
`experiencing premature cardiovascular events, such as heart attack or stroke.
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`There are no universally accepted criteria for the diagnosis of HoFH. Diagnosis is typically made clinically, using the following criteria:
` Assessment of cholesterol levels (TC or LDL-C);
`•
` Physical examination for the presence of xanthomas which are often present when HoFH is diagnosed in childhood, but may not be
`•
`present or noticeable when HoFH is diagnosed as an adult;
` An inadequate response to traditional lipid-lowering therapies that is not attributed to statin intolerance or to another identifiable
`cause;
` Assessment of the patient’s history with respect to premature atherosclerosis and cardiovascular disease; and
` Assessment of whether the family history of the patient on both sides is consistent with familial hypercholesterolemia (“FH”).
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`Genetic testing may be performed to make a diagnosis of HoFH, but is not routinely used in the U.S. because it is not widely available, and
`because genetic testing can fail to detect certain defects given the large number of possible mutations and the number of genes that could be
`involved, as described above. HoFH patients may have the same defect on both copies of the same gene or may have different defects, one
`inherited from each parent, on the same gene or defects inherited from each parent on two different genes each affecting the function of the
`LDL-R. A 2013 article in the European Heart Journal (“EHJ”) estimates that current genetic tests may fail to positively detect 10% to 40% of
`patients with FH. These estimates would suggest that any prevalence of HoFH based on genotyping, or extrapolated from genotyped
`heterozygous familial hypercholesterolemia (“HeFH”) patients, is underestimated. As a result, most physicians in the U.S. and in many other
`countries use clinical findings and family history on both sides to make a clinical diagnosis of HoFH. Although not widely used, HoFH may also
`be diagnosed through an assessment of LDL-R function in cultured skin fibroblasts.
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`Physicians treating patients with hypercholesterolemia, including HoFH, are highly focused on lowering levels of LDL-C in their patients.
`In the U.S., for example, organizations such as the National Cholesterol Education Program (“NCEP”), the American Heart Association, and the
`American College of Cardiology have emphasized aggressive management of LDL-C. NCEP guidelines currently recommend that patients at
`high risk of experiencing a heart attack achieve LDL-C levels of 100 mg/dL or lower through lifestyle changes and drug therapy as appropriate
`based on their starting levels. International guidelines for adult patients at high risk of experiencing a heart attack, such as those published in the
`International Journal of Cardiology and the Canadian Journal of Cardiology, and guidelines published in the EHJ in 2014 (2014 EHJ HoFH
`Guidelines) that are specific to HoFH support LDL-C treatment targets for such patients as low as 70 mg/dL. The American College of
`Cardiology and the American Heart Association released guidelines in 2013 for patients at high risk of cardiovascular disease caused by
`atherosclerosis that are focused first on lifestyle changes and statin therapy. The
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`2014 EHJ HoFH Guidelines made similar recommendations regarding lifestyle changes and statin therapy for the treatment of HoFH and also
`recommended the use of LDL apheresis, in which cholesterol is removed from the body through mechanical filtration, and the use of other
`adjunctive treatments, such as lomitapide and mipomersen, for HoFH patients who are within the indication for such products (adults for
`lomitapide). The clinical approach taken with HoFH patients has typically involved an aggressive treatment plan to reduce lipid levels as much
`as possible through dietary modifications and a combination of available lipid lowering drug therapies. Conventional drug therapies include
`statins, cholesterol absorption inhibitors and bile acid sequestrants. Less frequently, other drugs, such as niacin and fibrates, have been added to
`provide some incremental reductions in LDL-C levels, although these agents are typically used to modify lipids other than LDL-C. Because
`many of these therapies, including statins, act by increasing the activity of LDL-R, HoFH patients, given their impaired LDL-R function, or lack
`of function, often have an inadequate response to standard therapies. For example, high dose statin therapies that typically produce 46% to 55%
`reductions in LDL-C levels in the broad hypercholesterolemic patient population, on average, produce 14% to 30% reductions in patients with
`HoFH. Patients with HoFH who are unable to reach their recommended target LDL-C levels on drug therapy are sometimes treated using LDL
`apheresis. Although levels of LDL-C are reduced acutely using apheresis, there is a rapid rebound. Because apheresis provides only temporary
`reductions in LDL-C levels, it must be repeated frequently, typically one or two times per month. In addition, except in many countries in the
`EU, apheresis is not readily available, particularly in the U.S., due to the limited number of treatment centers that perform this procedure.
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`Lomitapide
`Mechanism of Action
`Lomitapide is a small molecule microsomal triglyceride transfer protein, or MTP, inhibitor, or MTP-I. MTP exists in both the liver and
`intestines where it plays a role in the formation of cholesterol. Given the fact that MTP is involved in the formation of cholesterol-carrying
`lipoproteins from both liver-related, or hepatic, and intestinal sources, we believe the inhibition of MTP makes an attractive target for
`cholesterol-lowering therapy.
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`Approval in the United States and European Union
`In December 2012, the FDA approved JUXTAPID as an adjunct to a low-fat diet and other lipid-lowering treatments, including LDL
`apheresis where available, to reduce LDL-C, TC, apo B and non-HDL-C in adult patients with HoFH. We launched JUXTAPID in the U.S. in
`late January 2013. The FDA has granted seven years of orphan drug exclusivity from the date of approval for JUXTAPID in the U.S. in the
`treatment of HoFH. The U.S. prescribing information for JUXTAPID specifies that the safety and effectiveness of lomitapide have not been
`established in patients with hypercholesterolemia who do not have HoFH or in pediatric patients, and that the effect of lomitapide on
`cardiovascular morbidity and mortality has not been determined.
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`In July 2013, we received marketing authorization for LOJUXTA in the EU as an adjunct to a low-fat diet and other lipid-lowering
`medicinal products with or without LDL apheresis in adult patients with HoFH. The Summary of Product Characteristics (“SmPC”) approved by
`the competent authorities for LOJUXTA describes that genetic confirmation of HoFH should be obtained whenever possible, and that other
`forms of primary hyperlipoproteinemia and secondary causes of hypercholesterolemia (e.g., nephrotic syndrome, hypothyroidism) must be
`excluded. The SmPC also specifies that the effect of lomitapide on cardiovascular morbidity and mortality has not been determined. We or our
`distributors have submitted documentation seeking pricing and reimbursement approvals from governmental authorities in key markets of the
`EU and government reimbursement approval actions are taking longer than previously anticipated. We anticipate reimbursement decisions in
`some of those countries in 2015. In March 2014, the G-BA deemed our dossier for LOJUXTA to be incomplete as a result of certain technical
`deficiencies. As a result of the technical deficiencies, LOJUXTA was automatically put into the category of “no additional benefit” under the G-
`BA process, without a review of the clinical merits, which limits the reimbursement level significantly. After the G-BA assessment, we withdrew
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`LOJUXTA from the German market in July 2014. We intend to re-file our dossier for LOJUXTA in June 2015, which we expect would result in
`an assessment by the G-BA in late 2015. In Italy, we are permitted to sell LOJUXTA on a named patient sales basis until the reimbursement and
`pricing approval process is completed in that country. LOJUXTA does not have orphan designation for the treatment of HoFH in the EU since
`the EMA considers the relevant condition for orphan designation purposes to include both HoFH and HeFH. We believe that the lack of orphan
`designation in the EU may be contributing to the extended time periods necessary to obtain reimbursement approval.
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`The prescribing information for lomitapide in the U.S. and the EU warns physicians that lomitapide can cause hepatotoxicity as manifested
`by elevations in transaminases and increases in hepatic fat, and that physicians are recommended to measure alanine aminotransferase (“ALT”),
`aspartate aminotransferase (“AST”), alkaline phosphatase, and total bilirubin before initiating treatment and then to measure ALT and AST
`regularly during treatment. During the first year of treatment, physicians must conduct a liver-related test prior to each increase in the dose of
`lomitapide or monthly, whichever occurs first. After the first year, physicians are required to perform these tests every three months and before
`increases in dose. The prescribing information in the EU provides further recommendations for monitoring for hepatic steatohepatitis/fibrosis
`and the risk of progressive liver disease, including annual imaging for tissue elasticity, and measuring of biomarkers and/or scoring methods in
`consultation with a hepatologist.
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`Because of the risk of liver toxicity, JUXTAPID is available in the U.S. only through a Risk Evaluation and Mitigation Strategy (“REMS”)
`program under which we certify all qualified healthcare providers who prescribe JUXTAPID and the pharmacies that dispense the medicine. The
`goals of the JUXTAPID REMS Program are:
` to educate prescribers about the risk of hepatotoxicity associated with the use of JUXTAPID and the need to monitor patients during
`•
`treatment with JUXTAPID as per product labeling; and
` to restrict access to therapy with JUXTAPID to patients with a clinical or laboratory diagnosis consistent with HoFH.
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`Similarly, in the EU, we have adopted risk management plans to help educate physicians on the safety information for LOJUXTA and
`appropriate precautions to be followed by healthcare professionals and patients.
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`Status outside the U.S. and the EU
`In early 2014, we received approval to market lomitapide as an adjunct treatment for adult patients with HoFH in Mexico and Canada.
`Lomitapide is also approved in Israel, Norway, Iceland, and Liechtenstein. The indications and prescribing information, including risk
`information, for lomitapide in Mexico, Canada and Israel are comparable to those in the U.S. The indications and prescribing information,
`including risk information, for lomitapide in Norway, Iceland, and Liechtenstein are comparable to those in the EU. Lomitapide is subject to a
`risk management plan in each country in which it is approved outside the U.S., except Israel, and such plans require the approval of regulatory
`authorities prior to reimbursement approval and marketing. The goal of the risk management plans is to help educate physicians on the safety
`information for lomitapide and appropriate precautions to be followed by healthcare professionals and patients. We have filed for marketing
`approval in certain additional countries, and expect to file for marketing approval in other countries where, in light of the potential size of the
`market and other relevant commercial and regulatory factors, it makes business sense to do so.
`
`We are also making lomitapide available in certain countries that allow use of a drug, on a named patient basis or under a compassionate
`use or other type of so-called expanded access program, before marketing approval has been obtained in such country. We charge for lomitapide
`for authorized pre-approval uses in some of the countries where it is available under an expanded access program, to the extent permitted by
`applicable law and local regulatory authorities. In 2014, the substantial majo