Aegerion Release: JUXTAPID (Lomitapide) Capsules Available On The Régie De l'Assurance Maladie Du Québec (RAMQ) For The Treatment Of Homozygous Familial Hypercholesterolemia (HoFH)

TORONTO, Nov. 20, 2015 (GLOBE NEWSWIRE) -- Aegerion Pharmaceuticals, Inc. (NASDAQ:AEGR), a biopharmaceutical company dedicated to the development and commercialization of innovative therapies for patients with debilitating rare diseases, today announced that the Régie de l'Assurance Maladie du Québec (RAMQ) has added JUXTAPID™ (lomitapide) capsules for the treatment of homozygous familial hypercholesterolemia (HoFH) to its formulary effective November 20, 2015. The listing was based on a recommendation from l'Institut National d'Excellence en Santé et en Services Sociaux (INESSS) and is subject to a Listing Agreement between the Quebec Minister of Health and Social Services and Aegerion Pharmaceuticals (Canada) Ltd. The Listing Agreement is based on Aegerion’s price for JUXTAPID in Canada, which is a fixed daily cost irrespective of the dose prescribed and strengths dispensed.

“We are pleased with decision of the RAMQ to add JUXTAPID to its formulary, furthering Aegerion’s goal of increasing access to our medicines for adult HoFH patients in need,” said Sandford D. Smith, Chief Executive Officer of Aegerion. “Canada is an important country in the development of Aegerion’s increasing global footprint.”

HoFH is a serious, rare genetic disease that impairs the function of the receptor responsible for removing LDL-cholesterol from the body, which results in extreme elevation of blood cholesterol levels. Patients with HoFH often develop premature and progressive atherosclerosis, a narrowing or blocking of the arteries.

“HoFH is a challenging disease, where patients continue to experience severe elevations in LDL-C despite the use of available therapies,” commented Dr. Jacques Genest, MD, FRCPC, Cardiologist at McGill University Health Centre, and Professor, Departments of Biochemistry, Medicine and Genetics at McGill University. "JUXTAPID has the ability to help reduce the cholesterol in adult HoFH patients and its approval on the RAMQ formulary is especially encouraging for the Quebec region, where there is a higher prevalence of HoFH based upon a founder effect.”

“FH Canada Patient Network knows first-hand the challenges of living with HoFH and applauds the Quebec Health Minister for accepting the INESSS recommendation to make this very important option available to patients with unmet needs,” added Durhane Wong-Rieger, Executive Director, FH Canada Patient Network.

The Regroupement Québécois des Maladies Orphelines (RQMO) has been representing patients with rare diseases in Quebec for more than 5 years. “It is comforting to see the Quebec government completing the full circle from assisting in bench side discovery to bedside availability of medicines. Having a medication like JUXTAPID funded for adult patients with HoFH breaks down the final barrier, and allows appropriate patients access to this innovative medicine,” said Gail Ouellette, geneticist, President and Executive Director of the RQMO.

About Aegerion Pharmaceuticals

Aegerion Pharmaceuticals is a biopharmaceutical company dedicated to the development and commercialization of innovative therapies for patients with debilitating rare diseases. Our first approved product, JUXTAPID, is an oral once-daily capsule that offers a treatment option to patients with homozygous familial hypercholesterolemia (HoFH) – a severe lipid disorder. For more information about the company, please visit www.aegerion.com.

Important Safety Information, including BOXED WARNING which states:

WARNING: RISK OF HEPATOTOXICITY

JUXTAPID can cause elevations in transaminases. In the JUXTAPID clinical trial, 10 (34%) of the 29 patients treated with JUXTAPID had at least one elevation in alanine aminotransferase (ALT) or aspartate aminotransferase (AST) =3x upper limit of normal (ULN). There were no concomitant clinically meaningful elevations of total bilirubin, international normalized ratio (INR), or alkaline phosphatase.

JUXTAPID also increases hepatic fat, with or without concomitant increases in transaminases. The median absolute increase in hepatic fat was 6% after both 26 and 78 weeks of treatment, from 1% at baseline, measured by magnetic resonance spectroscopy. Hepatic steatosis associated with JUXTAPID treatment may be a risk factor for progressive liver disease, including steatohepatitis and cirrhosis.

Measure ALT, AST, alkaline phosphatase, and total bilirubin before initiating treatment and then ALT and AST regularly as recommended. During treatment, adjust the dose of JUXTAPID if the ALT or AST are =3x ULN. Discontinue JUXTAPID for clinically significant liver toxicity.

Because of the risk of hepatotoxicity, JUXTAPID is available only through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS) called the JUXTAPID REMS PROGRAM.

CONTRAINDICATIONS

• Pregnancy

• Concomitant administration of moderate or strong CYP3A4 inhibitors

• Moderate or severe hepatic impairment or active liver disease including unexplained persistent elevations of serum transaminases

WARNINGS AND PRECAUTIONS

JUXTAPID can cause elevations in transaminases and hepatic steatosis. Although cases of hepatic failure have not been reported, there is concern that JUXTAPID could induce steatohepatitis, which can progress to cirrhosis over several years. Modify the dose of JUXTAPID if elevations of transaminases are observed and discontinue JUXTAPID for persistent or clinically significant elevations. If transaminase elevations are accompanied by clinical symptoms of liver injury, such as nausea, vomiting, abdominal pain, fever, jaundice, lethargy, flu-like-symptoms, increases in bilirubin =2x ULN, or active liver disease, discontinue treatment with JUXTAPID and identify the probable cause. Use JUXTAPID with caution when co-administered with agents known to be hepatotoxic. Alcohol may increase levels of hepatic fat and induce or exacerbate liver injury.

Measure ALT, AST, alkaline phosphatase, and total bilirubin before initiating treatment. During the first year, measure liver-related tests (ALT and AST, at a minimum) prior to each increase in dose or monthly, whichever occurs first. After the first year, do these tests at least every 3 months and before any increase in dose.

Females of reproductive potential should have a negative pregnancy test before starting JUXTAPID and should use effective contraception during therapy with JUXTAPID.

Given its mechanism of action in the small intestine, JUXTAPID may reduce the absorption of fat-soluble nutrients. Patients treated with JUXTAPID should take daily supplements that contain 400 international units vitamin E and at least 200 mg linoleic acid, 210 mg alpha-linolenic acid (ALA), 110 mg eicosapentaenoic acid (EPA), and 80 mg docosahexaenoic acid (DHA).

Gastrointestinal adverse reactions are common and may lead to treatment discontinuation. To reduce the risk of gastrointestinal adverse reactions, patients should adhere to a low-fat diet supplying less than 20% of energy from fat and the dosage of JUXTAPID should be increased gradually.

Combination with CYP3A4 inhibitors increases exposure to lomitapide. Strong and moderate CYP3A4 inhibitors should not be used with JUXTAPID. JUXTAPID dosage should not exceed 30 mg daily when used concomitantly with weak CYP3A4 inhibitors.

Due to risk of myopathy associated with simvastatin or lovastatin, doses of these agents should be limited when co-administered with JUXTAPID.

JUXTAPID increases the plasma concentrations of warfarin. Increases or decreases in the dose of JUXTAPID may lead to supra- or subtherapeutic anticoagulation, respectively. Patients taking warfarin should undergo regular monitoring of the INR, especially after any changes in JUXTAPID dosage.

Avoid use of JUXTAPID in patients with rare hereditary disorders of galactose intolerance.

ADVERSE REACTIONS

The most common adverse reactions were gastrointestinal, reported by 27 (93%) of 29 patients. Adverse reactions reported by =8 (28%) or more patients in the HoFH clinical trial included diarrhea, nausea, vomiting, dyspepsia and abdominal pain. Other common adverse reactions, reported by 5 to 7 (17-24%) patients, included weight loss, abdominal discomfort, abdominal distension, constipation, flatulence, increased ALT, chest pain, influenza, nasopharyngitis, and fatigue.

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