CAMBRIDGE, Mass.--(BUSINESS WIRE)--Genzyme Corporation (Nasdaq: GENZ) announced today that the FDA has
granted U.S. marketing approval for Lumizyme™ (alglucosidase alfa),
produced at the 4000 liter (L) bioreactor scale at its manufacturing
facility in Geel, Belgium. Lumizyme is the first treatment approved in
the United States specifically to treat patients with late-onset Pompe
“We are grateful to the
FDA for their efforts to approve Lumizyme ahead of its scheduled PDUFA
“This is an important day for the Pompe community, especially for those
patients with late-onset Pompe disease in the United States who are
awaiting treatment for this devastating disease,” said Genzyme Chairman
and Chief Executive Officer, Henri A. Termeer. “We are grateful to the
FDA for their efforts to approve Lumizyme ahead of its scheduled PDUFA
Lumizyme (alglucosidase alfa) is a lysosomal glycogen-specific enzyme
indicated for patients 8 years and older with late (non-infantile) onset
Pompe disease (GAA deficiency) who do not have evidence of cardiac
hypertrophy. The safety and efficacy of Lumizyme have not been evaluated
in controlled clinical trials in infantile-onset patients, or in late
(non-infantile) onset patients less than 8 years of age.
Genzyme began work on a therapy for Pompe disease ten years ago, and the
company has invested nearly $1 billion to support the development
program. In 2006, Genzyme received approval for Myozyme® (alglucosidase
alfa) in Europe and in other countries outside of the United States
manufactured at a 2000 L bioreactor scale and indicated to treat all
patients with Pompe disease. At this time, Genzyme also received FDA
approval for Myozyme manufactured at a smaller 160 L bioreactor scale in
the United States, which, because of its limited capacity, has been
reserved for children and infants in the United States. In 2009, Genzyme
received approval outside of the United States for manufacturing Myozyme
in 4000 L bioreactors at its state-of-the-art manufacturing facility in
Geel, Belgium, and began to transition patients globally to the product
manufactured at this larger scale. To prepare for growing demand for
alglucosidase alfa, Genzyme has installed a third 4000 L bioreactor in
Geel with an anticipated approval in 2011.
Genzyme has worked closely with patients and physicians in the U.S.
Pompe community during the preapproval period to assure that the most
severely affected late-onset patients could access therapy in advance of
Lumizyme approval. In May 2007, Genzyme began providing alglucosidase
alfa free-of-charge to patients in the United States through a program
known as the Alglucosidase Alfa Temporary Access Program (ATAP). Nearly
200 severely affected adults in the United States with Pompe disease are
currently receiving treatment under the ATAP program. Genzyme will now
work closely with the treating centers and prescribers to insure that
patients in the ATAP program can continue to access therapy during the
transition to commercial supply. Genzyme will also begin working with
U.S. healthcare professionals to help adult patients who have been
waiting to access treatment. In effort to preserve 160-L scale product
for infantile-onset patients, Genzyme will begin to transition eligible
patients who are receiving Myozyme onto Lumizyme.
Because Genzyme will market two approved alglucosidase alfa products in
the United States, a Risk Evaluation and Mitigation Strategy called the
Lumizyme ACE (alglucosidase alfa control and education) Program will be
implemented for Lumizyme to ensure appropriate use for the intended
patient populations. All prescribers of Lumizyme, and healthcare
facilities where Lumizyme will be dispensed and administered, are
required to be certified and enrolled in the Lumizyme ACE Program prior
to treating patients with Lumizyme. Prescribers must also ensure
patients enroll in the Lumizyme program prior to receiving therapy.
Genzyme will begin this process immediately to certify and enroll
prescribers and healthcare facilities and to help prescribers to enroll
all patients that they intend to treat with Lumizyme.
About Pompe Disease
Pompe Disease is a progressively debilitating disease that manifests as
a broad spectrum of clinical symptoms. All patients typically experience
progressive muscle weakness and breathing difficulty, but the rate of
disease progression can vary widely depending on the age of onset and
the extent of organ involvement. When symptoms appear within a few
months of birth, babies frequently display a markedly enlarged heart and
die within the first year of life. When symptoms appear during
childhood, adolescence or adulthood, patients may experience steadily
progressive debilitation and premature mortality due to respiratory
failure. They often require mechanical ventilation to assist with
breathing and wheelchairs to assist with mobility.
Important Safety Information about
Lumizyme has a boxed warning as follows: Life-threatening
anaphylactic reactions, severe allergic reactions and immune mediated
reactions have been observed in some patients during Lumizyme infusions.
Therefore, appropriate medical support should be readily available
when Lumizyme is administered.
Because of the potential risk of rapid disease progression in Pompe
disease patients less than 8 years of age, Lumizyme is available only
through a restricted distribution program called the Lumizyme ACE
Program. Only prescribers and healthcare facilities enrolled in the
program may prescribe, dispense, or administer Lumizyme. Lumizyme may be
administered only to patients who are enrolled in and meet all the
conditions of the Lumizyme ACE Program. To enroll in the Lumizyme ACE
Program call 1-800-745-4447.
Severe cutaneous reactions have been reported with alglucosidase alfa
including necrotizing skin lesions. Systemic immune mediated reactions,
including possible type III immune complex-mediated reactions have been
observed with alglucosidase alfa. Patients with acute underlying
respiratory illness or compromised cardiac and/or respiratory function
may be at risk of serious exacerbation of their cardiac or respiratory
compromise during infusions. Other serious adverse events that occurred
in a higher incidence in Lumizyme treated patients compared to placebo
included coronary artery disease, intervertebral disc protrusion,
pneumonia, gastroenteritis, and dehydration. The most common adverse
reactions observed in clinical studies were infusion reactions. Those
occurring in Lumizyme treated patients at an incidence of ≥ 5% compared
to placebo included anaphylaxis, urticaria, diarrhea, vomiting, dyspnea,
pruritus, rash/erythema, pharyngolaryngeal pain, neck pain, hypoacusis,
flushing/feeling hot, pain in extremity, fall and chest discomfort.
In post-marketing experience with Lumizyme, deaths, and serious adverse
reactions have been reported, including anaphylaxis. Adverse events
resulting in death reported in the postmarketing setting with Lumizyme
treatment included cardiorespiratory arrest, respiratory failure,
hemothorax, pneumothorax, cardiac failure, sepsis, aortic dissection,
cerebrovascular accident, and skin necrosis. The most frequently
reported serious adverse reactions were infusion reactions. The
following serious adverse events have been reported in at least 2
patients: dyspnea, respiratory failure, bronchospasm, stridor, decreased
oxygen saturation/hypoxia, pharyngeal edema, chest discomfort, chest
pain, hypotension, hypertension, erythema, flushing, lung infection,
tachycardia, cyanosis, and hypersensitivity. One case of
hyperparathyroidism has been reported.
here for the full prescribing information.
Important information safety information
Myozyme has a boxed warning as follows: Risk of Anaphylaxis
Life-threatening anaphylactic reactions have been observed in some
patients during Myozyme infusions. Therefore, appropriate medical
support should be readily available when Myozyme is administered.
Risk of Cardiorespiratory Failure
Patients with compromised cardiac or respiratory function may be at
risk of serious acute exacerbation of their cardiac or respiratory
compromise due to infusion reactions, and require additional monitoring.
Life-threatening and severe allergic reactions have included
anaphylactic shock, cardiac arrest, respiratory distress, hypotension,
bradycardia, hypoxia, bronchospasm, throat tightness, dyspnea,
angioedema, and urticaria. Acute cardiorespiratory failure requiring
intubation and inotropic support has been observed up to 72 hours after
infusion with Myozyme in infantile-onset Pompe disease patients with
underlying cardiac hypertrophy, possibly associated with fluid overload
with intravenous administration of Myozyme. Caution should be used when
administering general anesthesia for the placement of a central venous
catheter in infantile-onset Pompe disease patients with cardiac
hypertrophy. The most common adverse reactions requiring intervention
were infusion-related reactions which occurred in 20 of 39 (51%) of
patients treated with Myozyme in clinical studies. Some reactions were
here for the full prescribing information.
One of the world's leading biotechnology companies, Genzyme is dedicated
to making a major positive impact on the lives of people with serious
diseases. Since 1981, the company has grown from a small start-up to a
diversified enterprise with more than 12,000 employees in locations
spanning the globe and 2009 revenues of $4.5 billion.
With many established products and services helping patients in
approximately 100 countries, Genzyme is a leader in the effort to
develop and apply the most advanced technologies in the life sciences.
The company's products and services are focused on rare inherited
disorders, kidney disease, orthopaedics, cancer, transplant and immune
disease, and diagnostic testing. Genzyme's commitment to innovation
continues today with a substantial development program focused on these
fields, as well as cardiovascular disease, neurodegenerative diseases,
and other areas of unmet medical need.
Genzyme’s press releases and other company information are available at www.genzyme.com
and by calling Genzyme’s investor information line at 1-800-905-4369
within the United States or 1-678-999-4572 outside the United States.
This press release contains forward-looking statements regarding
Genzyme’s financial outlook and business plans including, without
limitation, statements about the availability of Lumizyme and the
potential expansion of manufacturing capacity for Myozyme and Lumizyme
in Geel, Belgium. These statements are subject to risks and
uncertainties that could cause actual results to differ materially from
those forecasted. These risks and uncertainties include, among others:
whether Genzyme has forecasted anticipated product demand accurately;
whether Genzyme is able to manufacture product in sufficient quantities
to meet demand; the timing of the implementation of the Lumizyme ACE
Program; whether the FDA and other regulatory authorities approve the
expansion of manufacturing capacity in Geel and the timing thereof; and
the risks and uncertainties described in Genzyme's SEC reports filed
under the Securities Exchange Act of 1934, including the factors
discussed under the caption "Risk Factors" in Genzyme's Amended
Quarterly Report on Form 10-Q/A for the quarter ended March 31, 2010.
Genzyme cautions investors not to place substantial reliance on the
forward-looking statements contained in this press release. These
statements speak only as of the date of this press release and Genzyme
undertakes no obligation to update or revise these statements.
Genzyme® and Myozyme® are registered trademarks,
and Lumizyme™ is a trademark, of Genzyme Corporation or its
subsidiaries. All rights reserved.
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