Sanofi Genzyme Begins Pivotal Phase 2/3 Trial of Olipudase Alfa for Adult Patients with Acid Sphingomyelinase Deficiency
July 06 2016 - 8:00AM
Business Wire
Sanofi Genzyme, the specialty care global business unit of
Sanofi, announced today that the first adult patient has enrolled
and been dosed in a pivotal Phase 2/3 clinical trial named ASCEND
for 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 (NPD B).
ASMD is one of a group of lysosomal storage disorders that
affect cellular metabolism and are caused by genetic mutations.
ASMD is a serious and life-threatening disorder caused by
insufficient activity of the enzyme acid sphingomyelinase resulting
in accumulation of sphingomyelin in multiple organs of the body.
Common clinical manifestations include enlarged liver and spleen,
liver dysfunction, infiltrative lung disease, bleeding
complications, cardiovascular and bone disease, and growth delay.
There are currently no approved treatment options for patients with
ASMD.
ASCEND is a Phase 2/3 multi-national, multi-center,
double-blinded, placebo-controlled trial to evaluate the efficacy,
safety, pharmacodynamics and pharmacokinetics of olipudase alfa
administered intravenously once every 2 weeks for 52 weeks in adult
patients with ASMD, specifically NPD B. The Phase 2/3 trial will
assess the effect of olipudase alfa on spleen size, lung function
and other important clinical parameters. Thirty-six patients are
expected to be enrolled in the study and receive olipudase alfa or
a placebo. Upon completion of the 52 week primary analysis period,
all patients will receive treatment in an extension period.
“ASMD is a rare and debilitating disease that can lead to
serious medical conditions including failure of the lungs, liver or
heart,” said Eugen Mengel, M.D., Principal Investigator at the
Villa Metabolica Mainz University Medical Center. “The beginning of
this pivotal trial is a critical milestone in the assessment of
olipudase alfa’s potential to impact the lives of patients living
with ASMD.”
In June of last year, Sanofi Genzyme announced the beginning of
a Phase 1/2 trial in pediatric patients with ASMD, specifically NPD
B. For more information on both trials, please visit
https://www.clinicaltrials.gov/ or
https://www.clinicaltrialsregister.eu. 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.
“We are excited to have clinical studies for olipudase alfa now
underway for both pediatric and adult patients,” said Therapeutic
Area Head, Rare Diseases Development Rand Sutherland, M.D. “This is
an important step forward in our journey to meet the need for a
meaningful treatment option for patients with ASMD. We are thankful
for the engagement and support of the patients, physicians and
scientific community for helping us reach this milestone.”
About SanofiSanofi, a global healthcare leader,
discovers, develops and distributes therapeutic solutions focused
on patients' needs. Sanofi is organized into five global business
units: Diabetes and Cardiovascular, General Medicines and Emerging
Markets, Sanofi Genzyme, Sanofi Pasteur and Merial.
Sanofi Genzyme focuses on developing specialty treatments for
debilitating diseases that are often difficult to diagnose and
treat, providing hope to patients and their families.
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Media RelationsSanofi GenzymeLisa Clemence, +1
617-768-6699lisa.clemence@genzyme.com
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