Genzyme,
a Sanofi company, announced today that the Food and Drug Administration
(FDA) has granted Fast Track designation for the development of
GZ/SAR402671, a new investigational oral substrate reduction therapy for
the treatment of Fabry disease.
FDA’s Fast Track Drug Development Program is designed to facilitate
frequent interactions with the FDA review team to expedite the clinical
development and review of a New Drug Application (NDA) for medicines
with the potential to treat serious or life-threatening conditions and
address unmet medical needs for such disease or conditions. It also
provides the opportunity to submit sections of an NDA on a rolling basis
before a sponsor submits the complete application. Genzyme is currently
enrolling patients in its Phase 2a trial of GZ/SAR402671, and plans to
enroll nine treatment-naïve male adult patients with Fabry disease in
this international, multicenter study.
Fabry disease is a rare lysosomal storage disorder that results in
abnormal tissue deposits of a particular fatty substance (called
globotriaosylceramide, also referred to as GL-3 or Gb3) throughout the
body. GZ/SAR402671 is a glucosylceramide synthase inhibitor that blocks
the formation of glucosylceramide (GL-1), a key intermediate in the
synthesis of GL-3.
“Becoming a Fast Track Program is an important milestone and we
appreciate this designation from FDA,” said Genzyme’s Acting Head of
Rare Diseases, Richard Peters, M.D, Ph.D. “We look forward to learning
more about this small molecule, with the goal of providing more
therapeutic options to the Fabry community as quickly as possible.”
About Fabry Disease
Fabry disease is an inherited and progressive condition that is
characterized by excessive accumulation of the lipid GL-3 in various
organs and tissues. Early symptoms include significant pain,
gastrointestinal disturbances, as well as other manifestations, and over
time, patients may experience life threatening renal, cardiac and
cerebrovascular events. As a result, patients with Fabry disease
typically have a shortened life span. Fabry disease affects both males
and females, with approximately 10,000 diagnosed patients in the world.
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.
Genzyme® is a registered trademark of Genzyme Corporation.
All rights reserved.
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 Genzyme. Sanofi
is listed in Paris (EURONEXT: SAN) and in New York (NYSE: SNY).
Forward Looking Statements
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the Private Securities Litigation Reform Act of 1995, as amended.
Forward-looking statements are statements that are not historical facts.
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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, 2014. Other than as required by applicable
law, Sanofi does not undertake any obligation to update or revise any
forward-looking information or statements.
Copyright Business Wire 2015