October 17, 2012
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`FDA Advisory Committee Recommends Approval of Lomitapide for Treatment of
`Homozygous Familial Hypercholesterolemia (HoFH)
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`CAMBRIDGE, Mass., Oct. 17, 2012 (GLOBE NEWSWIRE) -- Aegerion Pharmaceuticals, Inc. (Nasdaq:AEGR), an emerging
`biopharmaceutical company focused on the development and commercialization of novel therapeutics to treat debilitating and
`often fatal rare diseases, announced that the Endocrinologic and Metabolic Drugs Advisory Committee (EMDAC) of the U.S.
`Food and Drug Administration (FDA) determined by a vote of 13 to 2 that Aegerion has presented sufficient safety and efficacy
`data to support marketing of its product, lomitapide, for the treatment of patients with Homozygous Familial
`Hypercholesterolemia (HoFH) when used as an adjunct to a low-fat diet and other lipid-lowering therapies.
`
`"Today's Advisory Committee recommendation is an important milestone in our mission to provide patients with HoFH with
`urgently needed new treatment options to lower their cholesterol levels," said Marc Beer, Chief Executive Officer of Aegerion
`Pharmaceuticals. "We look forward to continuing to work closely with the FDA as the agency completes its review of our new
`drug application for lomitapide."
`
`The FDA has assigned a Prescription Drug User Fee Act (PDUFA) action date of December 29, 2012, for completion of its
`review of the New Drug Application (NDA) for lomitapide. The EMDAC provides the FDA with independent expert advice and
`recommendations. The FDA is not bound by the EMDAC's recommendation, but will consider the committee's recommendation
`as the FDA completes its review of the lomitapide NDA.
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`Conference Call Details
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`The Aegerion management team will hold a conference call with the investment community to discuss the outcome of the
`EMDAC meeting today at 7:00 pm EDT. To listen to the live conference call, dial (866) 516-3002 (International callers dial
`(760) 298-5082). In addition, the conference call will be available through a live audio webcast in the "Investors" section of the
`Aegerion website, www.aegerion.com. The conference call will be archived and accessible on the same website shortly after the
`conclusion of the call.
`
`About Lomitapide
`
`Lomitapide is a microsomal triglyceride transfer protein (MTP) inhibitor that Aegerion is developing as a once-day capsule for
`the treatment of patients afflicted with certain severe lipid disorders, including HoFH. MTP exists in both the liver and intestines
`where it plays a role in the formation of lipoproteins containing cholesterol and triglycerides. Inhibiting MTP reduces the level of
`cholesterol that the liver and intestines assemble and secrete into the bloodstream. Currently, there is no MTP inhibitor
`approved by the FDA for any indication.
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`Lomitapide has been evaluated in fourteen Phase I and eight Phase II clinical trials, as well as a pivotal Phase III clinical trial in
`HoFH completed in 2011. An extension study to assess long-term safety is ongoing. Over 900 patients have been treated with
`lomitapide as part of these clinical trials.
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`The most frequent adverse events in the Phase III clinical trial were gastrointestinal, and were generally mild to moderate.
`These events typically decreased after the patients were established on the maximally tolerated dose. Elevations in liver
`enzymes and hepatic fat were also observed in the Phase III trial. Four patients experienced elevations in liver enzymes of
`between five times to eleven times the upper limit of normal. Hepatic fat increased from a baseline of 1% to 8.3% at week 26,
`and then stabilized through week 78.
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`Aegerion submitted a NDA to the Food and Drug Administration (FDA), and a Marketing Authorization Application (MAA) to the
`European Medicines Agency (EMA), requesting approval to market lomitapide as an adjunct to a low-fat diet and other lipid-
`lowering therapies, with or without apheresis, to reduce LDL-C, total cholesterol, apolipoprotein B, and triglycerides in adults
`with HoFH.
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`About Homozygous Familial Hypercholesterolemia (HoFH)
`
`HoFH is a rare genetic lipid disorder that, if left untreated, results in extremely high cholesterol levels, typically between 400
`mg/dL and 1,000 mg/dL. Those affected are at severely high risk of experiencing premature cardiovascular events, such as
`heart attack or stroke, often experiencing their first cardiovascular event in their twenties. Despite current treatments, many
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`patients with HoFH do not survive beyond their mid-30's.
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`The disease is usually caused by defects in the low-density lipoprotein (LDL) receptor genes, resulting in impaired or total loss
`of function. The LDL receptor is a protein on the surface of cells that is responsible for binding to and removing cholesterol
`from the blood. A loss of LDL receptor function results in elevated accumulation of cholesterol in the blood.
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`About Aegerion Pharmaceuticals, Inc.
`
`Aegerion Pharmaceuticals, Inc.is an emerging biopharmaceutical company focused on the development and commercialization
`of novel, life-altering therapeutics to treat debilitating and often fatal rare diseases. The company's lead drug candidate,
`lomitapide, is in late-stage development for the treatment of Homozygous Familial Hypercholesterolemia (HoFH), a rare life-
`threatening disease characterized by severely elevated cholesterol levels. (cid:160)
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`Aegerion is motivated by its commitment to patients first, as well as its core values of integrity, innovation, responsibility to
`healthcare providers and development of employees, with a constant focus on scientific and clinical excellence. For more
`information, visit www.aegerion.com.
`
`Forward-Looking Statements
`
`This press release contains forward-looking statements, including statements regarding the potential for regulatory approval
`and launch of lomitapide. These forward-looking statements are neither promises nor guarantees of future performance, and
`are subject to a variety of risks and uncertainties, many of which are beyond our control, which could cause actual results to
`differ materially from those contemplated in these forward-looking statements. In particular, the risks and uncertainties include,
`among other factors:(cid:160)the risk that applicable regulatory authorities may ask for additional data, information or studies to be
`completed or provided prior to approval; the risk that the FDA may not follow the recommendations of EMDAC; the risk that
`applicable regulatory authorities may not agree with our validation plan or may require additional work related to the
`commercial manufacturing process to be completed prior to approval or may, in the course of the inspection of manufacturing
`facilities, identify issues to be resolved; the risks that the applicable regulatory authorities may not be satisfied with the safety
`profile of lomitapide; and the risk that we do not receive approval of lomitapide on a timely basis or at all. For additional
`disclosure regarding these and other risks we face, see the disclosure contained in our public filings with the U.S. Securities
`and Exchange Commission (available on the SEC's website at http://www.sec.gov), including the "Risk Factors" section of our
`(cid:160)most recent Quarterly Report on Form 10 -Q. We undertake no obligation to update or revise the information contained in this
`press release, whether as a result of new information, future events or circumstances or otherwise.
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`CONTACT: Aegerion Pharmaceuticals, Inc.
`
` Michael Lawless, VP, IR
`
` (857) 242-5028
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` mlawless@aegerion.com
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