Genzyme and Isis Provide Update on CHMP Opinion on KYNAMRO™ (mipomersen)

PR Newswire/Les Echos/ 
Genzyme and Isis Provide Update on CHMP Opinion on KYNAMRO(TM) (mipomersen) 
Cambridge, Mass. and Carlsbad, Calif., Dec. 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
"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 will continue to work with our colleagues at Genzyme
toward the marketing approval of KYNAMRO." 
An application of KYNAMRO is currently under review by the U.S. Food and Drug
Administration (FDA). In October 2012, KYNAMRO received a positive vote by the
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 Hypercholesterolemia (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 Hypercholesterolemia (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 
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 
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 
Isis Pharmaceuticals(r) is a registered trademark of Isis Pharmaceuticals, Inc.
Genzyme(r) is the registered trademark of Genzyme Corporation. All rights
KYNAMRO(TM) 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(TM) 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
Sanofi Media Relations                      Sanofi Investor Relations
Marisol Péron                               Sébastien Martel
Tel: +33 (0) 1 53 77 46 46                  Tel: +33 (0) 1 53 77 45 45
E-mail:                       E-mail: 
Genzyme Media Relations                     Sanofi Investor Relations
Ingrid Mitchell                             Kristen Galfetti
Tel: 617-768-6699                           Tel: +1 908 981 5560
E-mail:         E-mail: 
Media Contact Isis:
Amy Blackley, Ph.D.
760-603-2772 (Media) 
D. Wade Walke, Ph.D.
The content and accuracy of news releases published on this site and/or 
distributed by PR Newswire or its partners are the sole responsibility of the 
originating company or organisation. Whilst every effort is made to ensure the 
accuracy of our services, such releases are not actively monitored or reviewed 
by PR Newswire or its partners and under no circumstances shall PR Newswire or 
its partners be liable for any loss or damage resulting from the use of such 
information. All information should be checked prior to publication. 
-0- Dec/14/2012 12:58 GMT
Press spacebar to pause and continue. Press esc to stop.