Genzyme Initiates Phase 2a Clinical Trial to Evaluate Oral Therapy for Fabry Disease
April 21 2015 - 9:00AM
Business Wire
Genzyme, a Sanofi company, announced today that patients have
begun treatment in a Phase 2a clinical trial focused on evaluating
the pharmacodynamics of GZ/SAR402671, a new oral substrate
reduction therapy being investigated for the treatment of Fabry
disease. The safety, tolerability and pharmacokinetics of
GZ/SAR402671 will also be assessed. Genzyme 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.
The Phase 2a trial is an open label, single-arm clinical trial.
Patients will receive GZ/SAR402671 orally, once daily. The primary
endpoint in the Phase 2a trial will assess the ability of
GZ/SAR402671 to reduce accumulated GL-3 from the skin capillary
endothelium. Upon completion of the 26 week study, patients will
have the option to enroll into an extension study.
“We are excited to learn more about the potential of
GZ/SAR402671, which has a different mechanism of action than the
current standard of treatment for Fabry disease, enzyme replacement
therapy, and a potential ability to cross the blood brain barrier,”
said Genzyme’s Acting Head of Rare Diseases, Richard Peters, M.D,
Ph.D. “The ongoing efforts on this program underscore our long
standing commitment to develop innovative therapies intended to
address the unmet needs of the Fabry patient community.”
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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Media Contact: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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