FDA Grants Fast Track Designation to Genzyme’s Investigational Substrate Reduction Therapy for the Treatment of Fabry Disease
April 28 2015 - 01:00AM
Business Wire
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).
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Genzyme Media RelationsLori Gorski +1
617-768-9344lori.gorski@genzyme.comorSanofi Investor
RelationsSébastien Martel, +33 (0) 1 53 77 45 43ir@sanofi.com
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