FDA Grants Breakthrough Therapy Designation for Genzyme’s Olipudase Alfa
June 04 2015 - 1:00AM
Business Wire
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 U.S. Food
and Drug Administration (FDA) has granted Breakthrough Therapy
designation to olipudase alfa. This enzyme replacement therapy is
being investigated for the treatment of patients with
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), which results
in toxic accumulation of sphingomyelin. There are currently no
approved treatment options for patients with Niemann-Pick disease
type B.
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.
Olipudase alfa is being developed by Genzyme to potentially
address the fundamental defect underlying the disease.
Supplementing the defective or deficient native enzyme with
olipudase alfa allows for the breakdown of sphingomyelin, whose
accumulation is responsible for the clinical manifestation of
ASMD.
The Breakthrough Therapy designation is supported by data from a
completed Phase 1b study of olipudase alfa. Findings in five adult
patients with nonneuronopathic ASMD were presented at the Lysosomal
Disease Network’s WORLD Symposium in February 2015. The data
presented on the repeat-dose safety, pharmacodynamics, and
exploratory efficacy of olipudase alfa support its continued
development for the investigational use in nonneurological
manifestations of ASMD.
The company has started enrollment of a Phase 1/2 pediatric
study and is preparing for enrollment of a Phase 2/3 adult study in
the second half of 2015. For more information please visit
http://clinicaltrials.gov.
“There is tremendous unmet need in the ASMD/Niemann-Pick disease
type B community, and we are hopeful that olipudase alfa can be
developed into a meaningful treatment for patients,” said Genzyme’s
Global Head of Rare Diseases, Richard Peters, MD, Ph.D. “We
appreciate FDA’s support for this important program giving us the
opportunity to utilize an important expedited drug development
pathway for olipudase alfa and providing hope for patients affected
with a chronic and progressively debilitating disease.”
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 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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version on businesswire.com: http://www.businesswire.com/news/home/20150603006716/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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