Genzyme, a Sanofi company, today announced that it has received
the 2015 Industry Innovation Award from the National Organization
for Rare Disorders (NORD) in recognition of Cerdelga® (eliglustat)
capsules. Genzyme was selected for the company’s work in bringing
an additional treatment option to patients with Gaucher disease.
Cerdelga is the only first-line oral therapy for certain adult
Gaucher disease type 1 patients.
“Cerdelga is an important option for people living with Gaucher
disease, and underscores Genzyme’s commitment to understand and
respond to the needs of patients living with a rare disease,”
said Peter L. Saltonstall, President and CEO, NORD. "We
applaud Genzyme’s dedication to providing more choices to patients
in rare disease communities and are pleased to present them
with NORD’s 2015 Industry Innovation Award."
Genzyme has been researching an oral therapy for Gaucher disease
for fifteen years, from early chemistry and preclinical research
through clinical development. The Cerdelga clinical development
program is the largest ever conducted in Gaucher disease, with
approximately 400 patients treated in 29 countries.
"We are proud to receive this award. The approval of Cerdelga
exemplifies our commitment to bring innovative therapies forward to
improve patients’ lives through ongoing research,” said Genzyme’s
Head of Rare Diseases, Richard Peters, MD, Ph.D.
This award marks the fourth time that Genzyme has been
recognized by NORD for its contributions. The award will be
presented tonight at NORD’s "Portraits of Courage Celebration" in
Washington, DC.
The NORD Gala is an annual event at which researchers and others
are honored for improving the lives of people with rare diseases.
Each year NORD honors organizations that have made a positive
contribution to further the needs of the patient community, and
have inspired the public to do so as well.
NORD is a nonprofit organization established in 1983 to
represent all individuals and families affected by rare diseases.
It provides advocacy, education, support for research, and patient
assistance programs to improve the lives of the 30 million
Americans living with rare diseases.
About Gaucher DiseaseGaucher disease is an inherited
condition affecting fewer than 10,000 people worldwide. People with
Gaucher disease do not have enough of an enzyme, β-glucosidase
(glucocerebrosidase) that breaks down a certain type of fat
molecule. As a result, lipid engorged cells (called Gaucher cells)
amass in different parts of the body, primarily the spleen, liver
and bone marrow. Accumulation of Gaucher cells may cause spleen and
liver enlargement, anemia, excessive bleeding and bruising, bone
disease and a number of other signs and symptoms. The most common
form of Gaucher disease, type 1, generally does not affect the
brain.
About Genzyme’s Gaucher Disease ProgramDriven by a
long-term commitment to meeting unmet needs in the Gaucher
community, Genzyme has been researching and developing treatments
for those living with Gaucher disease for over 20 years. The
company’s first FDA-approved therapy was the world’s first
treatment for Gaucher disease. A few years later, Genzyme developed
a next-generation enzyme replacement therapy for Gaucher disease,
which is now the standard of care with unmatched years of efficacy
and safety data. In 2014, Genzyme was the first to bring to market
a first-line oral treatment for Gaucher disease, providing an
additional treatment option for patients. Genzyme will continue to
serve this community and remains committed to future
advancements.
About CerdelgaCerdelga (eliglustat), a novel
glucosylceramide analog given orally, was designed to partially
inhibit the enzyme glucosylceramide synthase, which results in
reduced production of glucosylceramide. Glucosylceramide is the
substance that builds up in the cells and tissues of people with
Gaucher disease. The concept was initially developed by the late
Norman Radin, PhD, from the University of Michigan. In pre-clinical
studies, the molecule, developed with James A. Shayman, MD, also
from the University of Michigan, showed specificity for
glucosylceramide synthase. Following an extensive pre-clinical and
early clinical research program, Cerdelga was studied in the
largest Phase 3 clinical program ever conducted in Gaucher
disease.
IMPORTANT SAFETY INFORMATION
Indications and UsageCERDELGA® (eliglustat) capsules are
indicated for the long-term treatment of adults with Gaucher
disease type 1 (GD1) who are CYP2D6 extensive metabolizers (EMs),
intermediate metabolizers (IMs), or poor metabolizers (PMs) as
detected by an FDA-cleared test. Patients who are CYP2D6
ultra-rapid metabolizers (URMs) may not achieve adequate
concentrations of CERDELGA to achieve a therapeutic effect. A
specific dose cannot be recommended for those patients whose CYP2D6
genotype cannot be determined (indeterminate metabolizers).
Important Safety InformationCERDELGA is contraindicated
in the following patients due to the risk of significantly
increased CERDELGA plasma concentrations which may result in
prolongation of the PR, QTc, and/or QRS cardiac intervals that
could result in cardiac arrhythmias: EMs or IMs taking a strong or
moderate CYP2D6 inhibitor concomitantly with a strong or moderate
CYP3A inhibitor and IMs or PMs taking a strong CYP3A inhibitor.
Drugs that inhibit CYP2D6 and CYP3A may significantly increase
the exposure to CERDELGA; Cerdelga dose adjustment may be needed,
depending on metabolizer status. See section 7 of the full
Prescribing Information for more details and other potentially
significant drug interactions.
Because CERDELGA is predicted to cause increases in ECG
intervals at substantially elevated plasma concentrations, use is
not recommended in patients with pre-existing cardiac disease, long
QT syndrome, or in combination with Class IA and Class III
antiarrhythmic medications.
The most common adverse reactions (≥10%) for CERDELGA are:
fatigue, headache, nausea, diarrhea, back pain, pain in
extremities, and upper abdominal pain.
Only administer CERDELGA during pregnancy if the potential
benefit justifies the potential risk; based on animal data,
CERDELGA may cause fetal harm. Discontinue drug or nursing based on
importance of drug to mother. CERDELGA is not recommended in
patients with moderate to severe renal impairment or in patients
with hepatic impairment.
To report SUSPECTED ADVERSE REACTIONS, contact Genzyme
Corporation at (1-800-745-4447) or FDA at 1-800-FDA-1088 or
www.fda.gov/medwatch.
Please see full Prescribing Information, including patient
Medication Guide, for additional important safety information.
Forward Looking StatementsThis press release contains
forward-looking statements as defined in the Private Securities
Litigation Reform Act of 1995, as amended. Forward-looking
statements are statements that are not historical facts. These
statements include projections and estimates and their underlying
assumptions, statements regarding plans, objectives, intentions and
expectations with respect to future financial results, events,
operations, services, product development and potential, and
statements regarding future performance. Forward-looking statements
are generally identified by the words "expects", "anticipates",
"believes", "intends", "estimates", "plans" and similar
expressions. Although Sanofi's management believes that the
expectations reflected in such forward-looking statements are
reasonable, investors are cautioned that forward-looking
information and statements are subject to various risks and
uncertainties, many of which are difficult to predict and generally
beyond the control of Sanofi, that could cause actual results and
developments to differ materially from those expressed in, or
implied or projected by, the forward-looking information and
statements. These risks and uncertainties include among other
things, the uncertainties inherent in research and development,
future clinical data and analysis, including post marketing,
decisions by regulatory authorities, such as the FDA or the EMA,
regarding whether and when to approve any drug, device or
biological application that may be filed for any such product
candidates as well as their decisions regarding labelling and other
matters that could affect the availability or commercial potential
of such product candidates, the absence of guarantee that the
product candidates if approved will be commercially successful, the
future approval and commercial success of therapeutic alternatives,
the Group's ability to benefit from external growth opportunities,
trends in exchange rates and prevailing interest rates, the impact
of cost containment policies and subsequent changes thereto, the
average number of shares outstanding as well as those discussed or
identified in the public filings with the SEC and the AMF made by
Sanofi, including those listed under "Risk Factors" and "Cautionary
Statement Regarding Forward-Looking Statements" in Sanofi's annual
report on Form 20-F for the year ended December 31, 2014. Other
than as required by applicable law, Sanofi does not undertake any
obligation to update or revise any forward-looking information or
statements.
About Genzyme, a Sanofi CompanyGenzyme 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.
About SanofiSanofi, 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).
Genzyme® and Cerdelga® are registered trademarks of Genzyme
Corporation. All rights reserved.
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version on businesswire.com: http://www.businesswire.com/news/home/20150519006133/en/
Genzyme Media RelationsIngrid Mitchell,
617-768-6699Ingrid.Mitchell@genzyme.com
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