Company Website:
http://www.genzyme.com
CAMBRIDGE, Mass. -- (Business Wire)
Genzyme,
a Sanofi company, announced today that the Ministry of Health, Labor and
Welfare (MHLW) has granted marketing approval for Cerdelga®
(INN: eliglustat), the only oral therapy for the treatment of adults
with Gaucher disease type 1 in Japan. Cerdelga was approved by the U.S.
Food and Drug Administration in August 2014, the European Commission in
January 2015, and the Australian Therapeutics Goods Administration in
February 2015.
The MHLW approval was based on data from the Cerdelga clinical
development program, the largest clinical research program ever
conducted in Gaucher disease type 1, with approximately 400 patients
treated in 29 countries. The development program included three Phase 3
clinical trials. In the Phase 3 treatment-naïve, placebo-controlled
trial, ENGAGE, improvements were seen across the following endpoints
after 9 months on Cerdelga: spleen size, platelet levels, hemoglobin
levels, and liver volume. The second Phase 3 trial, ENCORE, was designed
to assess disease stability in patients previously treated with enzyme
replacement therapy. That trial met the pre-specified criteria for
non-inferiority to an enzyme replacement therapy (imiglucerase), which
was a composite endpoint of the following parameters: spleen volume,
hemoglobin levels, platelet counts, and liver volume.
The third Phase 3 trial, EDGE, included 10 Japanese patients, and was
designed to evaluate different dosing frequencies of Cerdelga (in
patients previously treated with enzyme replacement therapy or treatment
naïve Gaucher disease patients). The EDGE trial included an interim
analysis of efficacy and safety in Japanese patients. Patients in all
three Phase 3 studies continue to receive Cerdelga in the extension
periods, and the majority of patients are in their 4th or 5th
year of treatment.
“The clinical development program for Cerdelga represents a global
effort across the entire Gaucher community, and we are grateful for
Japan’s efforts to provide access to Cerdelga to patients in their
country,” said Genzyme’s Acting Head of Rare Diseases, Richard Peters,
MD, Ph.D. “The program demonstrates Genzyme’s long-term commitment to
this disease community, and we recognize the collaborative efforts of
the physician and patient communities as the drivers that have made this
progress possible.”
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, acid 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 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
proposed by the late Norman Radin, PhD, from the University of Michigan.
In pre-clinical studies, the precursor molecule, developed with James A.
Shayman, MD, also from the University of Michigan, showed specificity
for glucosylceramide synthase. Following Genzyme’s extensive compound
optimization, pre-clinical and early clinical development program,
Cerdelga was studied in the largest Phase 3 clinical program ever
conducted in Gaucher disease, with approximately 400 patients treated in
30 countries.
IMPORTANT SAFETY INFORMATION (for US Healthcare Professionals)
Indications and Usage
CERDELGA® (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.
For full prescribing information and more information about Cerdelga for
U.S. patients, please visit: http://www.cerdelga.com/pdf/cerdelga_prescribing_information.pdf.
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.
Genzyme®, Cerezyme®, and Cerdelga® are
registered trademarks of Genzyme Corporation. All rights reserved.
About Sanofi
Sanofi, a global 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 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 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.
Contacts:
Genzyme Media Contact:
Lori Gorski, 617-768-9344
lori.gorski@genzyme.com
or
Sanofi
Investor Relations Contact:
Sebastien Martel, +33 (0) 1.53.77.45.45
IR@sanofi.com
Source: Genzyme
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