Aegerion Pharmaceuticals' JUXTAPID (lomitapide) Capsules Approved in Canada for the Treatment of Homozygous Familial Hypercholesterolemia (HoFH)
CAMBRIDGE, Mass., Feb. 10, 2014 (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 Health Canada has granted a Notice of Compliance (NOC) approving
JUXTAPID as an adjunct to a low-fat diet and other lipid-lowering
drugs, with or without LDL apheresis, to reduce low-density lipoprotein
cholesterol (LDL-C) in adult patients with homozygous familial
hypercholesterolemia (HoFH).
HoFH is a serious, rare genetic disease that impairs the function of
the receptor responsible for removing LDL-C ("bad" cholesterol) from
the body. A loss of LDL receptor function results in extreme elevation
of blood cholesterol levels. HoFH patients often develop premature and
progressive atherosclerosis, a narrowing or blocking of the arteries.
"We expect Canada to be an important market for Aegerion, and with
infrastructure established, we look forward to providing JUXTAPID to
HoFH patients in need of therapy," said Marc Beer, Chief Executive
Officer of Aegerion. "Approval in Canada marks another key milestone in
our plan for global expansion."
"HoFH is a challenging disease, and despite previously available
therapies such as statins, patients continue to experience severe
elevations in LDL-C," said Dr. Robert Hegele, MD, Western University
and London Health Sciences Centre. "The approval of JUXTAPID is an
especially encouraging event for the province of Quebec, where there is
a higher prevalence of HoFH based upon a founder effect," said Dr.
Jacques Genest, MD, McGill University.
Based on the risk of liver toxicity, JUXTAPID is subject to a risk
management plan in Canada. Due to its benefit-risk profile, the
prescribing of JUXTAPID should be limited to physicians experienced in
the diagnosis and treatment of familial hypercholesterolemia.
The safety and effectiveness of JUXTAPID have not been established in
patients with hypercholesterolemia who do not have HoFH, or in
pediatric patients. The effect of JUXTAPID on cardiovascular morbidity
and mortality has not been determined.
Important Safety Information from U.S. Prescribing 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,
increases in bilirubin greater than or equal to 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.
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(TM), 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 or www.aegerion.ca.
Forward-Looking Statements
This press release contains forward-looking statements, including
statements regarding the availability of JUXTAPID and launch of
JUXTAPID in Canada, the potential for JUXTAPID as a treatment for HoFH,
and our expectations with regard to the Canadian market. 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: the risk that reimbursement of JUXTAPID
may not be approved by government or other payers in Canada at levels
that we plan to seek or at all; the risk that JUXTAPID may not gain
market acceptance; and the risk that restrictions imposed by regulatory
authorities or the side effect profile may limit the commercial
potential of JUXTAPID. 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 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.
CONTACT: Investor/Media Contact:
Aegerion Pharmaceuticals, Inc.
Amanda Murphy, Manager of IR/PR
(857) 242-5024
[email protected]