CAMBRIDGE, Mass. & CARLSBAD, Calif.--(BUSINESS WIRE)--Genzyme, a Sanofi company (EURONEXT: SAN and NYSE: SNY), and Isis
Pharmaceuticals Inc. (NASDAQ: ISIS), announced today that the U.S. Food
and Drug Administration (FDA) has accepted for filing the New Drug
Application (NDA) for KYNAMRO™ (mipomersen sodium) for the
treatment of patients with homozygous familial hypercholesterolemia
(HoFH). The NDA filing with the FDA triggers a $25 million milestone
payment to Isis from Genzyme.
“We look forward to continuing the review process with the U.S.
and EU regulatory authorities to bring KYNAMRO™ to patients
in need.”
"The NDA filing with the FDA represents a significant achievement in the
development of KYNAMRO™ and our efforts to get this
important new drug to the market for patients who are at high-risk of a
cardiovascular event,” said David Meeker, M.D., President and CEO,
Genzyme. “We look forward to continuing the review process with the U.S.
and EU regulatory authorities to bring KYNAMRO™ to patients
in need.”
Genzyme submitted an application for U.S. marketing approval of KYNAMRO™
for the treatment of patients with HoFH in March 2012. The application
will be subject to a standard review and will have a Prescription Drug
User Fee Act (PDUFA) date of January 29, 2013. In July 2011, Genzyme
submitted an application for EU marketing approval of KYNAMROTM
for the treatment of patients with HoFH and severe heterozygous FH
(Severe HeFH).
“We believe that KYNAMRO™ has the potential to bring real
benefit to patients in the United States with HoFH who are unable to
adequately control their LDL-C with currently available treatments,”
said B. Lynne Parshall, Chief Operating Officer and CFO of Isis. “The
successes of our joint development efforts for KYNAMRO™ are
evident in the significant progress made in bringing this important new
drug to the regulatory agencies for review. We are pleased to have
earned the first regulatory milestone payment for KYNAMRO™ from
this collaboration.”
The FDA submission for KYNAMRO™ is supported by the largest
clinical trial conducted to date in the HoFH patient population. In the
randomized, double-blind, placebo controlled, multi-center trial,
significant reductions were observed in all atherogenic lipoproteins
evaluated (including LDL-C, Apo B and Lp(a)) for patients receiving
KYNAMRO™ who are already receiving a regimen of maximally
tolerated lipid lowering therapies including statins. Three patients (12
percent) treated with KYNAMRO™ withdrew due to adverse events.
Consistent with other studies evaluating KYNAMRO™, commonly
observed adverse events included mild to moderate injection site
reactions and flu-like symptoms, as well as elevations in liver
transaminases.
KYNAMRO™ is the registered trade name submitted to health authorities
for investigational agent mipomersen.
About KYNAMROTM (mipomersen sodium)
KYNAMROTM is a first-in-class apo-B synthesis inhibitor
currently in late-stage development for patients with homozygous
familial hypercholesterolemia (HoFH) and severe heterozygous familial
hypercholesterolemia (Severe HeFH) 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. KYNAMROTM 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, KYNAMROTM,
following regulatory approval, which is expected in 2012. 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® and KYNAMRO™ 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 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.