Submission Seeks Approval for Mipomersen’s Use in the Treatment of
Homozygous Familial Hypercholesterolemia and Severe Heterozygous
Familial Hypercholesterolemia
CAMBRIDGE, Mass. & CARLSBAD, Calif.--(BUSINESS WIRE)--Genzyme,
a Sanofi company (EURONEXT: SAN and NYSE: SNY), and Isis Pharmaceuticals
Inc. (NASDAQ: ISIS) today announced that Genzyme has submitted a
marketing authorization application (MAA) to the European Medicines
Agency seeking approval for the 200 mg weekly dose of mipomersen for the
treatment of homozygous and severe heterozygous familial
hypercholesterolemia.
“We also look forward to
our upcoming U.S. regulatory submission later this year, as these
submissions move us closer to our goal of making mipomersen available to
patients who are in the greatest need of new treatments.”
“This MAA submission is another significant step in the development of
mipomersen,” said Vice President and General Manager of Genzyme’s
Cardiovascular Business, Paula Soteropoulos. “We also look forward to
our upcoming U.S. regulatory submission later this year, as these
submissions move us closer to our goal of making mipomersen available to
patients who are in the greatest need of new treatments.”
Genzyme and Isis also announced today that, if the necessary approvals
are granted, mipomersen would be marketed under the brand name Kynamro®,
the registered name that has been submitted to health authorities for
the investigational agent.
“Mipomersen has the potential to change the management of patients with
homozygous and severe heterozygous familial hypercholesterolemia,” said
Chairman and CEO of Isis Pharmaceuticals, Stanley T. Crooke. “We are
excited by the progress we are making with Genzyme on this important
development program, which demonstrates the promise of antisense
technology to meet unmet medical needs.”
About Mipomersen
Mipomersen is a first-in-class apo-B synthesis inhibitor currently in
late-stage development for the reduction of LDL cholesterol (LDL-C). It
is intended to reduce LDL-C by preventing the formation of atherogenic
lipoproteins, the particles that carry cholesterol through the
bloodstream. Mipomersen acts by blocking the production of
apolipoprotein B (apoB), the protein that provides the structural core
for these atherogenic particles, including LDL and lipoprotein-a (Lp(a)).
About Familial Hypercholesterolemia
FH is a genetic disease that results in elevated LDL-C levels and family
patterns of increased risk of premature heart disease and heart
disease-related death. FH patients have inherited abnormalities in liver
cells that are responsible for clearing LDL particles from the blood. FH
is autosomal dominant, which means that all first-degree relatives of FH
patients have a 50 percent chance of having the disease as well, making
early detection through family screening critically important.
The most severe FH patients have LDL-C levels that are two to four times
higher than recommended levels, even when taking multiple
cholesterol-lowering medications. These people, who are characterized as
having severe FH, include: those who have inherited the disease from
both parents (homozygous FH (HoFH)) and those who have inherited it from
only one parent, and have a severe form of the disease (severe
heterozygous FH (severe HeFH)).
About Genzyme, a Sanofi Company
One of the world's leading biotechnology companies, Genzyme is dedicated
to making a major positive impact on the lives of people with serious
diseases. Since its founding in 1981, the company has introduced
breakthrough treatments that have provided new hope for patients. The
company’s areas of focus are rare genetic diseases, multiple sclerosis,
cardiovascular disease, and endocrinology. Genzyme is a Sanofi company.
Genzyme’s press releases and other company information are available at www.genzyme.com.
About Isis Pharmaceuticals, Inc.
Isis is exploiting its expertise in RNA to discover and develop novel
drugs for its product pipeline and for its partners. The Company has
successfully commercialized the world's first antisense drug and has 24
drugs in development. Isis' drug development programs are focused on
treating cardiovascular, metabolic, and severe and
rare/neurodegenerative diseases and cancer. Isis' partners are
developing antisense drugs invented by Isis to treat a wide variety of
diseases. Isis and Alnylam Pharmaceuticals are joint owners of Regulus
Therapeutics Inc., a company focused on the discovery, development and
commercialization of microRNA therapeutics. Isis also has made
significant innovations beyond human therapeutics resulting in products
that other companies, including Abbott, are commercializing. As an
innovator in RNA-based drug discovery and development, Isis has designed
and executed a patent strategy that has provided the Company with strong
and extensive protection for Isis’ drugs and technology. Additional
information about Isis is available at www.isispharm.com.
Sanofi Forward Looking Statement
This press release contains forward-looking statements as defined in the
Private Securities Litigation Reform Act of 1995, as amended.
Forward-looking statements are statements that are not historical facts.
These statements include projections and estimates and their underlying
assumptions, statements regarding plans, objectives, intentions and
expectations with respect to future financial results, events,
operations, services, product development and potential, and statements
regarding future performance. Forward-looking statements are generally
identified by the words “expects”, “anticipates”, “believes”, “intends”,
“estimates”, “plans” and similar expressions. Although Sanofi’s
management believes that the expectations reflected in such
forward-looking statements are reasonable, investors are cautioned that
forward-looking information and statements are subject to various risks
and uncertainties, many of which are difficult to predict and generally
beyond the control of Sanofi, that could cause actual results and
developments to differ materially from those expressed in, or implied or
projected by, the forward-looking information and statements. These
risks and uncertainties include among other things, the uncertainties
inherent in research and development, future clinical data and analysis,
including post marketing, decisions by regulatory authorities, such as
the FDA or the EMA, regarding whether and when to approve any drug,
device or biological application that may be filed for any such product
candidates as well as their decisions regarding labeling and other
matters that could affect the availability or commercial potential of
such products candidates, the absence of guarantee that the products
candidates if approved will be commercially successful, the future
approval and commercial success of therapeutic alternatives, the Group’s
ability to benefit from external growth opportunities as well as those
discussed or identified in the public filings with the SEC and the AMF
made by Sanofi, including those listed under “Risk Factors” and
“Cautionary Statement Regarding Forward-Looking Statements” in Sanofi’s
annual report on Form 20-F for the year ended December 31, 2010. Other
than as required by applicable law, Sanofi does not undertake any
obligation to update or revise any forward-looking information or
statements.
Isis Forward Looking Statement
This press release includes forward-looking statements regarding Isis’
collaboration with Genzyme, a sanofi company, and the development,
activity, therapeutic and safety of mipomersen in treating patients with
high cholesterol. Any statement describing Isis’ goals, expectations,
financial or other projections, intentions or beliefs, including the
planned commercialization of mipomersen is a forward-looking statement
and should be considered an at-risk statement. Such statements are
subject to certain risks and uncertainties, particularly those inherent
in the process of discovering, developing and commercializing drugs that
are safe and effective for use as human therapeutics, and in the
endeavor of building a business around such drugs. Isis’ forward-looking
statements also involve assumptions that, if they never materialize or
prove correct, could cause its results to differ materially from those
expressed or implied by such forward-looking statements. Although Isis’
forward-looking statements reflect the good faith judgment of its
management, these statements are based only on facts and factors
currently known by Isis. As a result, you are cautioned not to rely on
these forward-looking statements. These and other risks concerning Isis’
programs are described in additional detail in Isis’ annual report on
Form 10-K for the year ended December 31, 2010 and its most recent
quarterly report on Form 10-Q, which are on file with the SEC. Copies of
these and other documents are available from the Company.