Company Website:
http://www.genzyme.com
CAMBRIDGE, Mass. -- (Business Wire)
Genzyme,
a Sanofi company (EURONEXT: SAN and NYSE: SNY), today announced that the
U.S. Food and Drug Administration (FDA) approved a supplement to expand
the indication for Lumizyme® (alglucosidase alfa). Lumizyme
manufactured at the 4000L scale is now indicated for all Pompe patients
of any age or phenotype. The approval of this indication is now
consistent with that of the rest of the world, where alglucosidase alfa
manufactured at the 4000L is the only scale available. Previously, in
the United States, Lumizyme had been approved only for patients with
late onset Pompe disease.
“We are thankful to the entire Pompe community who has been on this
journey to provide a sustainable, long-term option for Pompe patients in
the United States,” said Genzyme President and CEO, David Meeker, M.D.
“We are pleased we can now offer alglucosidase alfa produced at the
4000L scale to all patients in the US.”
In the United States, alglucosidase alfa is manufactured at two
different production scales. Alglucosidase alfa manufactured at the 160L
scale (initial pilot scale) has a brand name of Myozyme®
(alglucosidase alfa)and alglucosidase alfa manufactured at
the 4000L (final manufacturing scale) has a brand name of Lumizyme.
Based on the biochemical and clinical data provided as part of the
submission, FDA concluded that alglucosidase alfa manufactured at both
scales in the US (i.e., Lumizyme and Myozyme) are comparable. The
overall safety profile of alglucosidase alfa remains unchanged.
Specific updates to the Lumizyme product label include:
-
Updated Indication: LUMIZYME® (alglucosidase alfa) is a
hydrolytic lysosomal glycogen-specific enzyme indicated for patients
with Pompe disease (GAA deficiency).
-
Inclusion of safety and efficacy data from infantile-onset studies
-
Removal of the REMS program
-
Update to the boxed warning to include infantile-onset specific
warning regarding fluid overload.
-
Updated to Pregnancy Category C classification
Lumizyme, marketed as Myozyme outside of U.S., is approved in more than
65 countries. For the complete Lumizyme label, visit www.lumizyme.com.
About Pompe Disease
Pompe disease is a progressive, debilitating and often fatal
neuromuscular disease caused by a genetic deficiency or dysfunction of
the lysosomal enzyme acid alpha-glucosidase (GAA) affecting an estimated
1 in 40,000 people worldwide. This enzymatic defect results in the
accumulation of glycogen primarily in muscle tissues that leads to
muscle weakness, loss of respiratory function, and often premature
death. Absent treatment, when symptoms occur in infancy, babies
typically die within the first year of life. When symptoms occur in
childhood or adulthood, patients often lose their ability to walk and
require wheelchairs to assist with mobility and experience difficulty
breathing as well as mechanical ventilation to breathe.
Important Safety Information for Lumizyme and
Myozyme
INDICATION
LUMIZYME® (alglucosidase alfa) is a hydrolytic lysosomal
glycogen-specific enzyme indicated for patients with Pompe disease (acid
α-glucosidase (GAA) deficiency).
IMPORTANT SAFETY INFORMATION
WARNING:RISK OF ANAPHYLAXIS, HYPERSENSITIVITY AND
IMMUNE-MEDIATED REACTIONS, and RISK OF CARDIORESPIRATORY FAILURE
See full prescribing information for complete boxed warning.
Life-threatening anaphylactic reactions and severe hypersensitivity
reactions, presenting as respiratory distress, hypoxia, apnea, dyspnea,
bradycardia, tachycardia, bronchospasm, throat tightness, hypotension,
angioedema (including tongue or lip swelling, periorbital edema, and
face edema), and urticaria, have occurred in some patients during and
after alglucosidase alfa infusions.Immune-mediated reactions
presenting as proteinuria, nephrotic syndrome, and necrotizing skin
lesions have occurred in some patients following alglucosidase alfa
treatment.Closely observe patients during and after
alglucosidase alfa administration and be prepared to manage anaphylaxis
and hypersensitivity reactions. Inform patients of the signs and
symptoms of anaphylaxis, hypersensitivity reactions, and immune-mediated
reactions and have them seek immediate medical care should signs and
symptoms occur.
Infantile-onset Pompe disease patients with compromised cardiac or
respiratory function may be at risk of serious acute exacerbation of
their cardiac or respiratory compromise due to fluid overload, and
require additional monitoring.
WARNINGS AND PRECAUTIONS
Anaphylaxis and Hypersensitivity Reactions:
Life-threatening anaphylaxis and hypersensitivity reactions have been
observed in some patients during and after treatment with alglucosidase
alfa. If anaphylaxis or severe hypersensitivity reactions occur,
immediately discontinue infusion and institute appropriate medical
treatment.
Immune-Mediated Reactions: Monitor patients
for the development of systemic immune-mediated reactions involving skin
and other organs.
Risk of Acute Cardiorespiratory Failure:
Patients with compromised cardiac or respiratory function may be at risk
of acute cardiorespiratory failure. Caution should be exercised when
administering alglucosidase alfa to patients susceptible to fluid volume
overload.
Risk of Cardiac Arrhythmia and Sudden Cardiac
Death during General Anesthesia for Central Venous Catheter Placement:
Caution should be used when administering general anesthesia for the
placement of a central venous catheter intended for alglucosidase alfa
infusion.
Appropriate medical support and monitoring measures should be available
during infusion.
ADVERSE REACTIONS
The most frequently reported adverse reactions (≥ 5%) in clinical trials
were hypersensitivity reactions and included: anaphylaxis, rash,
pyrexia, flushing/feeling hot, urticaria, headache, hyperhidrosis,
nausea, cough, decreased oxygen saturation, tachycardia, tachypnea,
chest discomfort, dizziness, muscle twitching, agitation, cyanosis,
erythema, hypertension/increased blood pressure, pallor, rigors, tremor,
vomiting, fatigue, and myalgia (6.1).
USE IN SPECIFIC POPULATIONS
Pregnancy: Based on animal data, alglucosidase alfa may cause fetal harm.
To report SUSPECTED ADVERSE REACTIONS, contact Genzyme at
1-800-745-4447 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch
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®, Myozyme®, and Lumizyme® are
registered trademarks of Genzyme Corporation. All rights reserved.
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).
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, 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
Ingrid Mitchell, 617-768-6699
Ingrid.Mitchell@genzyme.com
Source: Genzyme Corporation
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