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.
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Genzyme Media Contact:Lori Gorski,
617-768-9344lori.gorski@genzyme.comorSanofi Investor Relations
Contact:Sebastien Martel, +33 (0) 1.53.77.45.45IR@sanofi.com
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