Only First-Line Oral Therapy Approved for the Treatment of Adults
with Gaucher Disease Type 1
Company Website:
http://www.genzyme.com
CAMBRIDGE, Mass. -- (Business Wire)
Genzyme,
a Sanofi company (EURONEXT: SAN and NYSE: SNY), announced today that the
U.S. Food and Drug Administration (FDA) has approved Cerdelga™
(eliglustat) capsules, the only first-line oral therapy for certain
adult Gaucher disease type 1 patients. A small number of adult patients
who metabolize Cerdelga more quickly or at an undetermined rate, as
detected by an established genetic laboratory test, will not be eligible
for Cerdelga treatment. Cerdelga is expected to be available to patients
within a month.
“Cerdelga is an important new option for people living with Gaucher
disease type 1,” said Rhonda Buyers, CEO of the National Gaucher
Foundation. “As enzyme replacement therapy is the standard of treatment
for Gaucher disease, patients receive regular intravenous infusions for
life. With FDA's approval of a first-line oral treatment, Cerdelga has
the potential to be a valuable treatment option for people living with
this serious disease.”
“The approval of Cerdelga is encouraging for Gaucher patients because it
is a first-line oral treatment that has demonstrated a positive
risk/benefit profile, making it important from both a scientific and a
clinical perspective,” said Dr. Pramod Mistry, MD, PhD, Professor of
Medicine and Pediatrics and the Director of National Gaucher Disease
Treatment Center at Yale University School of Medicine. “While enzyme
replacement therapies break down fatty deposits that build up in cells
and cause a variety of symptoms, Cerdelga inhibits the accumulation of
these fatty deposits in the first place.”
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.
“More than twenty years ago, Genzyme introduced the world’s first
treatment for Gaucher disease. We are proud to build on this legacy and
continue to improve Gaucher patients’ lives through ongoing research and
new therapies,” said Genzyme President and CEO, David Meeker, M.D. “The
approval of Cerdelga represents our unwavering commitment to the Gaucher
patient community.”
The FDA approval was based on efficacy data from two positive Phase 3
studies for Cerdelga: one in patients new to therapy (Trial 1), and the
other in patients switching from approved enzyme replacement therapies
(Trial 2). The filing also incorporated four years of efficacy data from
the Cerdelga Phase 2 study.
In Trial 1, improvements were seen across the following endpoints after
9 months on Cerdelga: spleen size, platelet levels, hemoglobin levels,
and liver volume. Patients continue to receive Cerdelga in the extension
period, and the majority of patients have been on treatment for over
eighteen months.
Trial 2 met the pre-specified criteria for non-inferiority to an enzyme
replacement therapy (imiglucerase), which was a composite endpoint of
each of the following parameters: spleen volume, hemoglobin levels,
platelet counts, and liver volume. Patients continue to receive Cerdelga
in the extension period, and the majority of patients have been on
treatment for over two years.
The most common adverse reactions (≥10%) are fatigue, headache, nausea,
diarrhea, back pain, pain in extremities, and upper abdominal pain.
Cerdelga is a specific ceramide analogue inhibitor of glucosylceramide
synthase (IC50 = 10 ng/mL) with broad tissue distribution. It
reduces the production of glucosylceramide, the substance that builds up
in the cells and tissues of people with Gaucher disease. For more
information, visit
cerdelga.com.
See full
prescribing information for more details about warnings and
precautions and a complete list of adverse reactions.
Genzyme’s Case Managers provide individualized support services for
Gaucher patients, including helping patients identify insurance options;
referrals to financial assistance programs; assistance with treatment
authorizations if necessary; assistance with appeals; and access to
educational resources to help patients learn more about their disease.
To reach a local Genzyme Case Manager, call 800-745-4447 (press option
#3) Monday through Friday, from 8 a.m. to 6 p.m. ET, or visit our
Patient & Caregiver Support Services online portal at www.genzymesupportservices.com.
Marketing applications for Cerdelga are under review by the European
Medicines Agency (EMA) and other regulatory authorities.
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
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 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.
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 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, 2013. Other than as required by applicable
law, Sanofi does not undertake any obligation to update or revise any
forward-looking information or statements.
Contacts:
Media Contact:
Genzyme
Lori
Gorski, 617-768-9344
Lori.Gorski@genzyme.com
Source: Genzyme
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