CAMBRIDGE, Mass. & CARLSBAD, Calif. -- December 14, 2012 -- Genzyme, a Sanofi company (EURONEXT: SAN and NYSE: SNY), and Isis
Pharmaceuticals Inc. (NASDAQ: ISIS), today announced that the Committee for
Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA)
has adopted a negative opinion for its marketing authorization application
(MAA) for KYNAMRO^TM (mipomersen) for the treatment of patients with
Homozygous Familial Hypercholesterolaemia (HoFH). Genzyme plans to request a
re-examination of the CHMP Opinion.
"We are disappointed by the Committee's recommendation. Patients with HoFH
carry extreme, ongoing cardiovascular risk with significantly elevated LDL-C
levels despite use of currently available therapies,” said David Meeker,
President and CEO, Genzyme. “This is a rare disease patient population, with a
life-threatening condition, in need of new therapies. We will work closely
with the CHMP during the re-examination process to address the Committee's
concerns, with the goal of making this important medication available to HoFH
patients in Europe."
“We believe that we have generated significant evidence in support of
KYNAMRO,” said B. Lynne Parshall, Chief Operating Officer and CFO of Isis.
“Patients are in need of new options and we will continue to work with our
colleagues at Genzyme toward the marketing approval of KYNAMRO.”
An application for KYNAMRO is currently under review by the U.S. Food and Drug
Administration (FDA). In October 2012, KYNAMRO received a positive vote by an
FDA advisory panel that Genzyme had provided sufficient efficacy and safety
data to support the marketing of KYNAMRO for the treatment of patients with
Homozygous Familial Hypercholesterolaemia (HoFH).
About KYNAMRO (mipomersen)
KYNAMRO is a first-in-class apo-B synthesis inhibitor currently under
regulatory review for patients with homozygous familial hypercholesterolaemia
(HoFH) to further reduce LDL cholesterol (LDL-C) in patients already
maintaining a stable regimen of maximally-tolerated lipid-lowering therapies,
and who require additional significant lipid-lowering therapy. It is intended
to reduce LDL-C by preventing the formation of atherogenic lipoproteins, the
particles that carry cholesterol through the bloodstream. KYNAMRO acts by
blocking the production of apolipoprotein B (apo B), the protein that provides
the structural core for these atherogenic particles, including LDL and
lipoprotein-a (Lp(a)).
About Familial Hypercholesterolaemia (FH)
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 (HoFH) and those who
have inherited it from only one parent, and have a particularly severe form of
the disease (Severe HeFH) defined as those people who are maximally treated
and still have LDL-C greater than 200 mg/dL (5.1 mmol) with coronary heart
disease or greater than 300 mg/dL (7.1 mmol) without coronary heart disease.
People with HoFH may have aggressive heart disease beginning in childhood, and
even with today’s therapies remain at significant risk of cardiovascular
events. Learn more at www.FHJourneys.com.
About Genzyme, a Sanofi Company
Genzyme has pioneered the development and delivery of transformative therapies
for patients affected by rare and debilitating diseases for over 30 years. We
accomplish our goals through world-class research and with the compassion and
commitment of our employees. With a focus on rare diseases and multiple
sclerosis, we are dedicated to making a positive impact on the lives of the
patients and families we serve. That goal guides and inspires us every day.
Genzyme’s portfolio of transformative therapies, which are marketed in
countries around the world, represents groundbreaking and life-saving advances
in medicine. As a Sanofi company, Genzyme benefits from the reach and
resources of one of the world’s largest pharmaceutical companies, with a
shared commitment to improving the lives of patients. Learn more at
www.genzyme.com.
About Sanofi
Sanofi, a global and diversified healthcare leader, discovers, develops and
distributes therapeutic solutions focused on patients’ needs. Sanofi has core
strengths in the field of healthcare with seven growth platforms: diabetes
solutions, human vaccines, innovative drugs, consumer healthcare, emerging
markets, animal health and the new Genzyme. Sanofi is listed in Paris
(EURONEXT: SAN) and in New York (NYSE: SNY).
About Isis Pharmaceuticals, Inc.
Isis is exploiting its leadership position in antisense technology to discover
and develop novel drugs for its product pipeline and for its partners. Isis'
broad pipeline consists of 25 drugs to treat a wide variety of diseases with
an emphasis on cardiovascular, metabolic and severe and rare/neurodegenerative
diseases, and cancer. Isis' partner, Genzyme, plans to commercialize Isis'
lead product, KYNAMRO, following regulatory approval. Isis' patents provide
strong and extensive protection for its drugs and technology. Additional
information about Isis is available at www.isispharm.com.
Isis Pharmaceuticals^® is a registered trademark of Isis Pharmaceuticals, Inc.
Genzyme^® is the registered trademark of Genzyme Corporation. All rights
reserved.
KYNAMRO™ is the registered trademark of Genzyme Corporation submitted to
health authorities for investigational agent mipomersen. All rights reserved.
Sanofi Forward Looking Statements
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 labelling and other matters that could affect the availability or
commercial potential of such product candidates, the absence of guarantee that
the product 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, trends in exchange
rates and prevailing interest rates, the impact of cost containment policies
and subsequent changes thereto, the average number of shares outstanding 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, 2011. 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 benefit and safety of KYNAMRO™ in treating patients with high
cholesterol. Any statement describing Isis’ goals, expectations, financial or
other projections, intentions or beliefs, including the planned
commercialization of KYNAMRO, 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, 2011 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.
Contact:
Sanofi Media Relations
Marisol Péron, +33 (0) 1 53 77 46 46
mr@sanofi.com
or
Genzyme Media Relations
Ingrid Mitchell, 617-768-6699
Ingrid.Mitchell@genzyme.com
or
Media Contact Isis:
Amy Blackley, Ph.D., 760-603-2772 (Media)
or
D. Wade Walke, Ph.D., 760-603-2741 (Investors)
or
Sanofi Investor Relations
Sébastien Martel, +33 (0) 1 53 77 45 45
ir@sanofi.com
or
Kristen Galfetti, +1 908-981-5560
ir@sanofi.com