CAMBRIDGE, Mass. & CARLSBAD, Calif.--(BUSINESS WIRE)--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.
“Patients are in need of new options and we will continue to
work with our colleagues at Genzyme toward the marketing approval of
KYNAMRO.”
"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.