Genzyme and Isis Provide Update on CHMP Opinion on KYNAMRO (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 KYNAMROTM (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 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 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.
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
Media Contact Isis:
Amy Blackley, Ph.D.
760-603-2772 (Media) D. Wade Walke, Ph.D.
760-603-2741(Investors)
Posted: December 2012
