Press Release: Xenpozyme® (olipudase alfa) approved in Japan, first
and only approved therapy indicated to treat acid sphingomyelinase
deficiency
Xenpozyme® (olipudase alfa) approved in Japan,
first and only approved therapy indicated to treat acid
sphingomyelinase deficiency
- Xenpozyme
represents first Sanofi therapy to be approved under the SAKIGAKE
‘fast-track’ designation
- Approval based on
positive results from two separate clinical trials in children and
adults demonstrating improvement in lung function (as measured by
DLco) and reduction of spleen and liver volumes
Paris, March
28, 2022. The Japanese Ministry of
Health, Labor, and Welfare (MHLW) has granted marketing
authorization for Xenpozyme® (olipudase alfa) for the treatment of
adult and pediatric patients with non-central nervous system
(non-CNS) manifestations of acid sphingomyelinase deficiency
(ASMD), a rare, progressive, and potentially life-threatening
genetic disease. Xenpozyme is currently the only approved treatment
for ASMD and represents Sanofi’s first therapy to be approved under
the SAKIGAKE (or “pioneer”) designation, which is the Japanese
government’s regulatory fast-track pathway to promote research and
development of innovative new medical products addressing urgent
unmet medical needs.
John Reed,
M.D., Ph.DExecutive Vice President,
Global Head of Research and Development, Sanofi“Today’s approval of
Xenpozyme is a watershed moment for ASMD patients and their
families, representing 20 years of research and the shared efforts
of advocacy partners, clinicians, and patients. As the world’s
first medicine approved for ASMD, Xenpozyme offers a potentially
transformative option for this historically neglected community. We
are proud of this achievement and grateful that Japan’s PDMA has
recognized the significance of the unmet need that Xenpozyme
addresses with the Sakigake designation. At Sanofi, we are working
with health authorities globally, including the EU where olipudase
alfa has PRIME designation and in the USA where this enzyme
replacement therapy has Breakthrough designation, to rush this
important medicine to ASMD patients around the world.”
Xenpozyme is a recombinant human acid
sphingomyelinase enzyme developed to replace deficient or defective
acid sphingomyelinase (ASM), an enzyme that allows for the
breakdown of the lipid sphingomyelin. Accumulation of sphingomyelin
in cells can cause harm to the lungs, spleen, and liver, as well as
other organs, potentially leading to early death.
The approval of Xenpozyme in Japan is based on
positive results from the ASCEND and ASCEND-Peds clinical trials,
showing that Xenpozyme provided improvement in lung function (as
measured by diffusing capacity of the lung for carbon monoxide, or
DLco) and reduction of spleen and liver volumes, with a
well-tolerated safety profile in adults and children with ASMD.
These data were presented at the American Society of Human
Genetics (ASHG) 2020 Virtual Meeting.
Xenpozyme has been evaluated in children and
adults to treat non-CNS manifestations of ASMD type A/B and ASMD
type B. Xenpozyme has not been studied in patients with ASMD type
A.
Historically known as Niemann-Pick disease types A,
A/B, and B, ASMD is a genetically-based, progressive, and
potentially life-threatening disease. ASMD represents a spectrum of
disease, with two types that may represent opposite ends of a
continuum referred to as ASMD type A and ASMD type B. ASMD type A/B
is an intermediate form that includes varying degrees of CNS
involvement. Until now, no approved therapies for ASMD have been
available anywhere in the world.
Outside of Japan, olipudase alfa is being
evaluated by regulatory authorities around the world. A Biologics
License Application (BLA) for olipudase alfa was accepted for
Priority Review by the U.S. Food and Drug Administration (FDA),
with a decision expected early Q3 2022. The European Medicines
Agency (EMA) has awarded olipudase alfa the PRIority MEdicines
(PRIME) designation, and a decision is anticipated in the second
half of 2022.
About SanofiWe are an innovative global
healthcare company, driven by one purpose: we chase the miracles of
science to improve people’s lives. Our team, across some 100
countries, is dedicated to transforming the practice of medicine by
working to turn the impossible into the possible. We provide
potentially life-changing treatment options and life-saving vaccine
protection to millions of people globally, while putting
sustainability and social responsibility at the center of our
ambitions. Sanofi is listed on EURONEXT: SAN and NASDAQ: SNY
Media RelationsSally
Bain | + 1 617 834 6026
| sally.bain@sanofi.com
Investor RelationsEva
Schaefer-Jansen | + 33 7 86 80 56 39
| eva.schaefer-jansen@sanofi.comArnaud
Delépine | + 33 6 73 69
36 93 | arnaud.delepine@sanofi.comCorentine
Driancourt | + 33 6 40 56 92 21 |
corentine.driancourt@sanofi.comFelix
Lauscher | + 1 908 612 7239 |
felix.lauscher@sanofi.comPriya
Nanduri | +1 908 981 5560 |
priya.nanduri@sanofi.com Nathalie
Pham | + 33 7 85 93 30 17 |
nathalie.pham@sanofi.com
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