“RegistryNXT!” builds upon largest resource in the world for Gaucher
disease data
CAMBRIDGE, Mass.--(BUSINESS WIRE)--Genzyme,
a Sanofi company (EURONEXT: SAN and NYSE: SNY), announced today the
launch of a new web-based platform, RegistryNXT!SM, to
support the International Collaborative Gaucher Group (ICGG) Gaucher
Registry. The first and largest database of its kind in the world, the
global ICGG Gaucher Registry was built to promote the understanding of
Gaucher and to track the outcomes of treatments worldwide. Now in its 20th
year, nearly 30 published papers on Gaucher disease have resulted from
the wealth of observational data in the registry.
“After twenty years of data collection and analysis, the fundamental
goals of the Gaucher Registry remain the same: to advance the medical
and scientific understanding of Gaucher disease and to improve the
quality of care of patients around the world”
Since 1991, Genzyme has worked closely with the international Gaucher
community to understand disease epidemiology, natural history and
long-term treatment outcomes. Because of the rarity of diseases like
Gaucher, having one central repository of data is crucial to understand
disease progression and to help manage patient outcomes. The ICGG
Gaucher Registry remains the only resource of its kind for Gaucher
disease, with over 700 participating physicians representing nearly
6,000 patients and over 40,000 patient-years of follow-up data. Genzyme
was the first company in the world to commercialize a treatment for
Gaucher disease, and today Cerezyme® (imiglucerase for injection) is the
standard of care for patients with Gaucher disease type 1.
“After twenty years of data collection and analysis, the fundamental
goals of the Gaucher Registry remain the same: to advance the medical
and scientific understanding of Gaucher disease and to improve the
quality of care of patients around the world,” said Genzyme’s Head of
Clinical Development and Medical Affairs, Richard Moscicki, MD.
“RegistryNXT! will facilitate real-time and more comprehensive analysis
of data, enabling publication of additional data and development of
evidence-based disease management guidelines.”
The launch of the RegistryNXT! application follows three years of
development, with new features that include interactive, real-time
report generation for disease management, more efficient data collection
and more opportunity for patient access to their information, with the
involvement of their treating physician. Plans are underway to roll out
this new Registry platform for Genzyme’s other lysosomal storage
disorder registries: Pompe, Fabry and Mucopolysaccharidosis I (MPS I).
“One of the most important accomplishments of the registry to date is
its direct impact on patient care through its ability to provide a solid
base of evidence for best clinical practices, including patient
assessments and recommendations on quantifiable treatment goals,” said
Neal J. Weinreb, MD, Chairman of the ICGG North American Board and
co-Chair of the ICGG International Board. “Over the years, patients have
repeatedly sought greater direct involvement with the Gaucher Registry.
Registry NXT! will now enable physicians to share real time data with
patients both at the point of service and via personal computers, a
major advance that should enhance patient involvement, understanding and
satisfaction.”
The ICGG Gaucher Registry is overseen by international and regional
boards of scientific advisors. These advisors are external physicians
that form the governance body, oversee the scientific integrity and
guide research, publications and policy. Genzyme has provided financial
support of the operations, estimated to be over $50 million since 1991.
The ICGG Gaucher Registry is operated by a team of individuals from
various functional areas at Genzyme, including medical directors, data
managers, statisticians, health outcomes researchers, epidemiologists
and programmers.
Cerezyme important safety information
Approximately 15 percent of patients have developed IgG antibodies, and
these patients have a higher risk of hypersensitivity reaction.
Therefore periodic monitoring is suggested; caution should be exercised
in patients with antibodies or prior symptoms of hypersensitivity.
Symptoms suggestive of hypersensitivity occurred in 6.6 percent of
patients, and include anaphylactoid reaction, pruritus, flushing,
urticaria, angioedema, chest discomfort, dyspnea, coughing, cyanosis and
hypotension. Reactions related to Cerezyme administration have been
reported in less than 15 percent of patients. Each of the following
events occurred in less than two percent of the total patient
population. Reported adverse events include nausea, vomiting, abdominal
pain, diarrhea, rash, fatigue, headache, fever, dizziness, chills,
backache and tachycardia. Adverse events associated with the route of
administration include discomfort, pruritus, burning, swelling or
sterile abscess at the site of venipuncture. For full prescribing
information, please visit www.genzyme.com.
About Genzyme, a Sanofi Company
One of the world's leading biotechnology companies, Genzyme is dedicated
to making a major positive impact on the lives of people with serious
diseases. Since its founding in 1981, the company has introduced
breakthrough treatments that have provided new hope for patients. The
company’s areas of focus are rare genetic diseases, multiple sclerosis,
cardiovascular disease, and endocrinology. Genzyme is a Sanofi company.
Genzyme’s press releases and other company information are available at www.genzyme.com.
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, rare
diseases, consumer healthcare, emerging markets and animal health.
Sanofi is listed in Paris (EURONEXT: SAN) and in New York (NYSE: SNY).