Genzyme Initiates Phase 1/2 Clinical Trial to Evaluate Olipudase Alfa in Pediatric Patients
June 15 2015 - 8:00AM
Business Wire
First patient dosed with investigational enzyme
replacement therapy designed to treat the nonneurological
manifestations of ASMD which characterize Niemann-Pick disease type
B
Genzyme, a Sanofi company, announced today that the first
pediatric patient has begun treatment in a Phase 1/2 clinical trial
focused on evaluating the investigational therapy olipudase alfa.
Olipudase alfa is an enzyme replacement therapy being studied for
the treatment of nonneurological manifestations of acid
sphingomyelinase deficiency (ASMD), also known as Niemann-Pick
disease type B, as opposed to type A, which is characterized by
neurological involvement. ASMD is a serious and life-threatening
disorder caused by insufficient activity of the enzyme acid
sphingomyelinase (ASM) resulting in toxic accumulation of
sphingomyelin. There are currently no approved treatment options
for patients with Niemann-Pick disease type B.
The Phase 1/2 trial is a multi-national, multi-center, open
label, ascending dose trial to evaluate the safety, tolerability
and pharmacokinetics of olipudase alfa administered intravenously
once every 2 weeks for 52 weeks in pediatric patients with ASMD.
Twelve pediatric patients will be enrolled into 3 age cohorts: an
adolescent cohort (12 to <18 years of age); a child cohort (6 to
<12 years of age); and an infant/early child cohort (birth to
<6 years of age). The primary objective of the Phase1/2 trial is
to assess the safety and tolerability of olipudase alfa. Upon
completion of the 52-week trial, patients will have the option to
enroll into an extension study. Genzyme is preparing for enrollment
to begin in a Phase 2/3 trial involving adult patients with ASMD in
the second half of 2015. For more information please visit
http://clinicaltrials.gov.
The U.S. Food and Drug Administration (FDA) has granted
Breakthrough Therapy designation to olipudase alfa. Breakthrough
Therapy designation is intended to expedite the development and
review of investigational new drugs that target serious or
life-threatening conditions. The criteria for granting Breakthrough
Therapy designation are preliminary clinical evidence of
substantial improvement on a clinically significant endpoint over
available therapies. The Breakthrough Therapy designation is
distinct from the FDA’s other mechanisms to expedite drug
development and review, and will allow for a close collaboration
between Genzyme and the FDA on the olipudase alfa development
program.
“With start of clinical studies both in pediatric and adult
patients just a few months apart, we are confirming our commitment
to the Niemann Pick disease patient community to advance the
development of this novel therapy as quickly as possible and for
both patient populations,” said Head of the Sanofi Genzyme R&D
Center, Jim Burns, Ph.D. “We are very grateful to the patients for
their engagement and their support in advancing this exciting
program.”
About ASMD (Niemann-Pick Disease)
Traditionally called Niemann-Pick disease types A and B (NPD A
and NPD B), Acid Sphingomyelinase Deficiency (ASMD) is one of a
group of lysosomal storage diseases that affect the metabolism and
that are caused by genetic mutations. ASMD is caused by the
deficiency of a specific enzyme, acid sphingomyelinase (ASM). This
enzyme is found in special compartments within cells called
lysosomes and is required to metabolize a lipid called
sphingomyelin. If ASM is absent or not functioning properly,
sphingomyelin cannot be metabolized properly and is accumulated
within the cell, eventually causing cell death and the malfunction
of major organ systems. Niemann-Pick A and Niemann-Pick B are both
caused by the same enzymatic deficiency and there is growing
evidence that the two forms represent opposite ends of a
continuum.
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 healthcare leader, discovers, develops and
distributes therapeutic solutions focused on patients’ needs.
Sanofi has core strengths in 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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defined in the Private Securities Litigation Reform Act of 1995, as
amended. Forward-looking statements are statements that are not
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among other things, the uncertainties inherent in research and
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future approval and commercial success of therapeutic alternatives,
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report on Form 20-F for the year ended December 31, 2014. Other
than as required by applicable law, Sanofi does not undertake any
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statements.
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version on businesswire.com: http://www.businesswire.com/news/home/20150615005479/en/
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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