Genzyme,
a Sanofi company, announced today that the Committee for Medicinal
Products for Human Use (CHMP) of the European Medicines Agency (EMA) has
adopted a positive opinion recommending approval of Cerdelga®
(eliglustat) capsules, an oral treatment for certain adults living with
Gaucher disease type 1.
The European Commission (EC) is expected to make a final decision on
granting marketing authorization for Cerdelga in the EU in the coming
months. Cerdelga was approved by the U.S. Food and Drug Administration
in August, and is under review by other regulatory authorities around
the world.
“Today’s CHMP opinion is the next step in ensuring that Cerdelga is
available to eligible adults with Gaucher disease type 1 living in the
European Union,” said David Meeker, MD, Genzyme President and CEO. “The
standard of care for Gaucher disease is Cerezyme® (imiglucerase for
injection). With the availability of an effective oral therapy, Cerdelga
would offer some patients and their physicians more choice in how to
manage this serious disease.”
The CHMP opinion was based on data from the Cerdelga clinical
development program, which is the largest ever conducted in Gaucher
disease, with approximately 400 patients treated in 29 countries.
Genzyme has been researching an oral therapy for Gaucher disease for
fifteen years.
The most common adverse reactions (greater than or equal to 10%) are
fatigue, headache, nausea, diarrhea, back pain, pain in extremities, and
upper abdominal pain.
About Gaucher Disease
Gaucher 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 Program
Driven 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 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 a new treatment option for patients. Genzyme will continue to
serve this community and remains committed to future advancements.
About Cerdelga
Cerdelga (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 Usage
CERDELGATM (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 Information
CERDELGA 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.
Cerezyme Important Safety Information
Approximately 15% of patients have developed IgG antibodies to Cerezyme
during the first year of therapy. Approximately 46% of patients with
detectable IgG antibodies experienced symptoms of hypersensitivity, and
these patients have a higher risk of hypersensitivity. It is suggested
that patients be monitored periodically for IgG antibody formation
during the first year of treatment.
Hypersensitivity has also been observed in patients without detectable
IgG antibodies. Symptoms suggestive of hypersensitivity have been noted
in approximately 6.6% of all patients, and anaphylactoid reactions in
less than 1%. Treatment with Cerezyme should be approached with caution
in patients who have exhibited hypersensitivity symptoms such as
pruritus, flushing, urticarial, angioedema, chest discomfort, dyspnea,
coughing, cyanosis, and hypotension. Pre-treatment with antihistamines
and/or corticosteroids and a reduced rate of infusion may allow
continued treatment in most patients.
In less than 1% of patients, pulmonary hypertension and pneumonia have
been observed during treatment with Cerezyme. These are known
complications of Gaucher disease regardless of treatment. Patients with
respiratory symptoms in the absence of fever should be evaluated for the
presence of pulmonary hypertension.
Approximately 13.8% of patients have experienced adverse events related
to treatment with Cerezyme. Some of these are injection site reactions
such as discomfort, pruritus, burning, swelling or sterile abscess at
the site at the site of venipuncture. Additional adverse reactions that
have been reported include nausea, abdominal pain, vomiting, diarrhea,
rash, fatigue, headache, fever, dizziness, chills, backache, and
tachycardia. Transient peripheral edema has also been reported for this
therapeutic class of drug.
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.
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 the new Genzyme. Sanofi is listed in Paris (EURONEXT:
SAN) and in New York (NYSE: SNY).
Forward Looking Statements
This 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
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future financial results, events, operations, services, product
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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, 2013. Other than as
required by applicable law, Sanofi does not undertake any obligation to
update or revise any forward-looking information or statements.
Copyright Business Wire 2014