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Genzyme and Isis Announce FDA Approval of KYNAMRO™ (mipomersen sodium) Injection for the Treatment of Homozygous Familial

PR Newswire/Les Echos/ 
PRESS RELEASE 


              Genzyme and Isis Announce FDA Approval of KYNAMRO(TM) 
     (mipomersen sodium) Injection for the Treatment of Homozygous Familial
                              Hypercholesterolemia

Paris, France and Carlsbad, California, January 29, 2013 - Sanofi (EURONEXT: 
SAN and NYSE: SNY) and its subsidiary Genzyme, and Isis Pharmaceuticals Inc.
(NASDAQ: ISIS), today announced that the U.S. Food and Drug Administration 
(FDA) has approved its New Drug Application (NDA) for KYNAMRO(TM) (mipomersen 
sodium) injection. KYNAMRO, given as a 200 mg weekly subcutaneous injection, 
has been approved as an adjunct to lipid-lowering medications and diet to 
reduce low density lipoprotein-cholesterol (LDL-C), apolipoprotein B (Apo B), 
total cholesterol (TC), and non-high density lipoprotein-cholesterol 
(non HDL-C) in patients with homozygous familial hypercholesterolemia (HoFH).

"Today's FDA appro val of KYNAMRO is great news for patients with HoFH who are
in need of additional treatment options for this rare, and often 
under-diagnosed disease," said Genzyme President and CEO David Meeker, M.D. 
"As the leader in treatments for rare diseases, we are pleased to bring our 
expertise to HoFH patients living with this serious condition to better help 
them manage their disease."

HoFH is a rare inherited condition that makes the body unable to remove LDL
cholesterol, often called the "bad" cholesterol, from the blood, causing
abnormally high levels of circulating LDL cholesterol. In the United States,
HoFH, an orphan indication, occurs in approximately one in one million
individuals. For those with HoFH, heart attacks and death often occur before 
age 30.

"People living with Homozygous FH do not appear to be sick, but they live with
the burden of this rare disease every day," said Katherine Wilemon, President
and Founder the FH Foundation. "The appro val of KYNAMRO gives the HoFH
community hope that HoFH can be effectively managed."

The FDA approval triggers a $25 million milestone payment to Isis from
Genzyme.

"KYNAMRO is the first systemic antisense drug to reach the market and is the
culmination of two decades of work to create a new, more efficient drug
technology platform. As evidenced by our robust pipeline, our antisense drug
discovery technology is applicable to many different diseases, including the
treatment of a chronic and rare disease, like HoFH," said Stanley T. Crooke,
M.D., Ph.D., Chairman of the Board and CEO of Isis. "We look forward to
continuing to work with Genzyme toward a successful commercial launch of 
KYNAMRO and global expansion into other markets."

The FDA approval for KYNAMRO is supported by the largest clinical trial
conducted to-date in the HoFH patient population. The randomized, double-blind,
placebo-controlled, multi-center trial enrolled 51 patients age 12 to 53 years,
including 7 patients age 12 to 16 years, who were maintaining a regimen of
maximally-tolerated lipid lowering medications. Treatment with KYNAMRO further
reduced LDL-C levels by an average of 113 mg/dL, or 25%, from a treated 
baseline of 439 mg/dL, and further reduced all measured endpoints for 
atherogenic particles. In March 2010, these data were published in The Lancet 
by Professor Raal, University of the Witwatersrand in South Africa.

Safety data for KYNAMRO are based on pooled results from four Phase 3,
randomized, double-blind, placebo-controlled trials with a total of 390 
patients of which 261 patients received a weekly subcutaneous injection of 200 
mg of KYNAMRO and 129 patients received placebo for a median treatment duration
of 25 weeks. Eighteen percent of patients on KYNAMRO and 2% of patients on 
placebo discontinued treatment due to adverse reactions. The most common 
adverse reactions in patients treated with KYNAMRO that led to treatment 
discontinuation and occurred at a rate greater than placebo were: injection 
site reactions (5.0%), alanine aminotransferase (ALT) increased (3.4%), 
flu-like symptoms (2.7%), aspartate aminotransferase (AST) increased (2.3%), 
and liver function test abnormal (1.5%).

KYNAMRO is an antisense drug is metabolized without affecting the CYP450
pathways used in commonly prescribed drugs, and thus has potential for no
drug-drug interactions. No clinically relevant pharmacokinetic interactions 
were reported between KYNAMRO and warfarin, or between KYNAMRO and simvastatin 
or ezetimibe.

KYNAMRO contains a Boxed Warning citing the risk of hepatic toxicity. Patients
taking KYNAMRO should have liver enzyme testing before starting the drug and
periodically thereafter. See below for Important Safety Information about
KYNAMRO.

The safety and effectiveness of KYNAMRO have not been established in patients
with hypercholesterolemia who do not have HoFH. The effect of KYNAMRO on
cardiovascular morbidity and mortality has not been determined.

Because of the risk of hepatotoxicity, KYNAMRO is available only through a Risk
Evaluation and Mitigation Strategy (REMS) called the KYNAMRO REMS. The goals of
the KYNAMRO REMS are:

  * To educate prescribers about the risk of hepatotoxicity associated with the
    use of KYNAMRO, and the need to monitor patients during treatment with 


KYNAMRO as per product labeling.
  * To restrict access to therapy with KYNAMRO to patients with a clinical or 
laboratory diagnosis consistent with homozygous familial  
hypercholesterolemia (HoFH). 
As part of its commitment to HoFH patients, Genzyme has developed KYNAMRO
CornerstoneSM, an HoFH and KYNAMRO support program for healthcare providers,
patients, and their families. KYNAMRO Cornerstone services include: 
* Dedicated KYNAMRO Cornerstone Case Managers.
  * Product & disease education for providers, patients, and families.
  * In-person injection training, if requested.
  * Reimbursement support, including out-of-pocket financial support, for 
patients who qualify.
  * Coordination of KYNAMRO shipment and delivery. 
KYNAMRO Cornerstone Case Managers are available live Monday-Friday from 9 am 
to 6 pm Eastern time. For more information about KYNAMRO Cornerstone, or about
these support services call 1-877- KYNAMRO (877-596-2676). For additional
information, please visit www.KYNAMRO.com. 
Important Safety Information  
WARNING: RISK OF HEPATOTOXICITY 
KYNAMRO can cause elevations in transaminases. In the KYNAMRO clinical trial in
patients with HoFH, 4 (12%) of the 34 patients treated with KYNAMRO compared
with 0% of the 17 patients treated with placebo had at least one elevation in
alanine aminotransferase (ALT) >3x upper limit of normal (ULN). There were no
concomitant clinically meaningful elevations of total bilirubin, international
normalized ratio (INR) or partial thromboplastin time (PTT). 
KYNAMRO also increases hepatic fat, with or without concomitant increases in
transaminases. In the trials in patients with heterozygous familial
hypercholesterolemia (HeFH) and hyperlipidemia, the median absolute increase in
hepatic fat was 10% after 26 weeks of treatment, from 0% at baseline, measured
by magnetic resonance imaging (MRI). Hepatic steatosis is a risk factor for
advanced liver disease; including steatohepatitis and cirrhosis. 
Measure ALT, AST, alkaline phosphatase, and total bilirubin before initiating
treatment and then ALT, AST regularly as recommended. During treatment, 
withhold the dose of KYNAMRO if the ALT or AST are >3 x ULN. Discontinue 
KYNAMRO for clinically significant liver toxicity. 
Because of the risk of hepatotoxicity, KYNAMRO is available only through a
restricted program under a Risk Evaluation and Mitigation Strategy (REMS) 
called the KYNAMRO REMS. 
OTHER WARNINGS AND PRECAUTIONS 
Patients are advised to read the KYNAMRO medication guide before starting
treatment with KYNAMRO, and each time they receive a refill. There may be new
information. This information does not take the place of talking to a doctor
about a medical condition or treatment. 
KYNAMRO may cause serious side effects, including liver problems. A doctor
should be informed of any liver problems, including liver problems while taking
other medicines, or if a patient has any of these symptoms of liver problems
while taking KYNAMRO: nausea, vomiting, fever, loss of appetite, being (or
feeling) more tired than usual, yellowing of eyes or skin, dark urine, itching,
or stomach pain. 
Alcohol may increase levels of hepatic fat and induce or exacerbate liver
injury. It is recommended that patients taking KYNAMRO should consume no more
than one alcoholic drink per day. 
Caution should be exercised when KYNAMRO is used with other medications known 
to have potential for hepatotoxicity. 
KYNAMRO should be used during pregnancy only if clearly needed. Females who
become pregnant during KYNAMRO therapy should notify their healthcare
provider. 
Safety and effectiveness have not been established in pediatric patients. 
KYNAMRO is not recommended in patients with severe renal impairment, clinically
significant proteinuria, or on renal dialysis. 
The safety and effectiveness of KYNAMRO as an adjunct to LDL apheresis have not
been established; therefore, the use of KYNAMRO as an adjunct to LDL apheresis
is not recommended. 
CONTRAINDICATIONS 
KYNAMRO is contraindicated in the following conditions: 
* Moderate or severe hepatic impairment (Child-Pugh B or C) or active liver 
disease, including unexplained persistent elevations of serum  
transaminases.
  * Patients with a known hypersensitivity to any component of this product. 
COMMON SIDE EFFECTS
In clinical trials the most commonly-reported adverse reactions were
injection site reactions occurring in 84% of patients receiving KYNAMRO versus
33% of placebo treated patients. The most common injection site reactions were
erythema (59%), pain (56%), hematoma (32%), pruritus (29%), swelling (18%) and
discoloration (17%). Injection site reactions did not occur with every 
injection but resulted in discontinuation of therapy in 5% of patients in 
pooled phase 3 trials.
Flu-like symptoms, defined as any one of the following: influenza-like
illness, pyrexia, chills, myalgia, arthralgia, malaise or fatigue and occurring
within 2 days of injection, have been reported more frequently in patients
receiving KYNAMRO (30%) versus placebo (16%) in the pooled Phase 3 trials.
Flu-like symptoms did not occur with all injections but resulted in
discontinuation of therapy in 3% of patients in pooled phase 3 trials. 
See full prescribing information for more details about Warnings & Precautions,
complete list of Adverse Reactions and Boxed Warning.  
About KYNAMRO (mipomersen sodium) injection
KYNAMRO is indicated as a first-in-class, oligonucleotide inhibitor, of
apolipoprotein B-100 synthesis. KYNAMRO is an adjunct to lipid-lowering
medications and diet to reduce low density lipoproteincholesterol (LDL-C),
apolipoprotein B (apo B), total cholesterol (TC), and non-high density
lipoproteincholesterol (non HDL-C) in patients with homozygous familial
hypercholesterolemia (HoFH). KYNAMRO reduces LDL-C by preventing the formation
of atherogenic lipoproteins, the particles that carry cholesterol through the
bloodstream. KYNAMRO acts by blocking the production of apo B, the protein that
provides the structural core for these atherogenic particles, including LDL. 
About Homozygous Familial Hypercholesterolemia (HoFH)
HoFH is a rare genetic disease characterized by extreme cholesterol levels.
People with HoFH have inherited mutations that limit the body's ability to 
clear cholesterol. HoFH is extremely rare: it is believed to occur in only one 
out of every one million persons. As with other rare diseases, the true 
prevalence of HoFH may be underestimated because of inadequate data and 
under-diagnosis. Today, it is estimated that HoFH affects about 6,000 people 
globally. Medical literature includes different criteria for marking an HoFH 
diagnosis. HoFH may be diagnosed by clinical or genetic parameters, and may be 
considered in cases of unusually high LDL-C, such as greater than 500 mg/dL 
without treatment, or 300 mg/dL after taking cholesterol-lowering medication. 
Because HoFH is genetic, it is important that all family members of people with
HoFH know their cholesterol levels, regardless of their age. 
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 28 drugs to treat a wide variety of diseases with an
emphasis on cardiovascular, metabolic, severe and rare diseases, and cancer.
Isis' partner, Genzyme, is commercializing Isis' lead product, KYNAMRO, in the
United States for the treatment of patients with HoFH. Genzyme is also pursuing
marketing approval of KYNAMRO in Europe. Isis' patents provide strong and
extensive protection for its drugs and technology. Additional information about
Isis is available at www.isispharm.com. 
Isis Pharmaceuticals(r) is a registered trademark of Isis Pharmaceuticals, Inc. 
Genzyme(r), KYNAMRO(TM) and KYNAMRO CornerstoneSM are registered trademarks of
Genzyme Corporation. 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 Statements
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 Company. 
Contacts:
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: mr@sanofi.com                  E-mail: ir@sanofi.com  
Genzyme Media Relations                Sanofi Investor Relations
Ingrid Mitchell                        Kristen Galfetti
Tel: 617-768-6699                      Tel: +1 908 981 5560
E-mail: Ingrid.Mitchell@genzyme.com    E-mail: ir@sanofi.com  
Isis Media Relations
Amy Blackley, Ph.D.
760-603-2772 (Media)
E-mail: ABlackley@isisph.com  
Isis Investor Relations
D. Wade Walke, Ph.D.
760-603-2741(I nvestors) 
E-mail: WWalke@isisph.com 
                  
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-0- Jan/30/2013 07:39 GMT
 
 
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