|[August 28, 2014]
Pfizer and Protalix BioTherapeutics Announce FDA Approval of Pediatric Indication for ELELYSO™ (taliglucerase alfa) for Injection, for Intravenous Use for the Treatment of Type 1 Gaucher Disease
NEW YORK & CARMIEL, Israel --(Business Wire)--
Pfizer Inc. (NYSE:PFE) and Protalix BioTherapeutics, Inc. (NYSE-MKT:PLX,
TASE:PLX) announced today that the U.S. Food and Drug Administration
(FDA) approved ELELYSO™ (taliglucerase alfa) for injection for pediatric
patients. ELELYSO is therefore now indicated for long-term enzyme
replacement therapy (ERT) for adult and pediatric patients with a
confirmed diagnosis of Type 1 Gaucher disease.
"The approval of ELELYSO to treat pediatric patients with Type 1 Gaucher
disease provides physicians another treatment option for this rare and
potentially debilitating disease," said Rory O'Connor, Senior Vice
President, Global Medical Affairs, Global Innovative Pharma Business,
Pfizer Inc. "This pediatric indication, along with the recent
announcement that ELELYSO received kosher certification by the Orthodox
Union (OU), reinforces the ongoing commitment of Pfizer to addressing
the needs of the Gaucher community."
The safety and efficacy of ELELYSO were assessed in fourteen pediatric
patients with Type 1 Gaucher disease in two clinical trials. The first
trial consisted of nine patients in a 12-month, multi-center,
double-blind, randomized study in treatment-naïve patients aged two to
13 years. At the end of the 12-month study, therapeutic efficacy of
ELELYSO was demonstrated, as measured by a decrease in spleen and liver
volume and an increase in platelet count. A second trial consisted of 5
pediatric patients aged 6 to 16 years who were switched from
imiglucerase to ELELYSO. The trial was a 9-month, multi-center,
open-label, single-arm study in patients who had been receiving
treatment with imiglucerase at dosages ranging from 9.5 units/kg to 60
units/kg every other week for a minimum of 2 years. ELELYSO was
administered for 9 months at the same dose as each patient's previous
imiglucerase dose. If needed, adjustment of dosage was allowed during
the study in order to maintain stability of clinical parameters. Mean
spleen and liver volume, platelet count and hemoglobin value remained
stable through 9 months of ELELYSO treatment.
The recommended dosage of ELELSYO for treatment-naïve adult and
pediatric patients four years of age and older is 60 units per kg of
body weight administered every other week as a 60 to 120 minute
Patients previously treated on a stable dosage of imiglucerase are
recommended to begin treatment with ELELYSO at that same dosage when
they switch from imiglucerase to ELELYSO. Dosage adjustments can be made
based on achievement and maintenance of each patient's therapeutic goals.
Serious hypersensitivity reactions, including anaphylaxis, have occurred
in some patients treated with ELELYSO. The most common adverse reactions
for ELELYSO in clinical trials were itching, flushing, headache, joint
pain, pain in extremity, abdominal pain, vomiting, fatigue, back pain,
dizziness, nausea and rash. Vomiting occurred more often in pediatric
patients than adults.
"While Type 1 Gaucher disease can manifest in childhood or adulthood,
the disease more often presents during childhood," said Paige Kaplan,
MB, BCh, Section of Biochemical Genetics (Metabolic Diseases),
Children's Hospital of Philadelphia. "It is important that children with
this disease have access to a range of FDA-approved treatment options
that are effective."
As part of its ongoing commitment to helping address the unmet medical
needs of people with rare diseases, Pfizer also offers a specialized
support program -- called Gaucher Personal Support (GPS) -- for people
living with Gaucher disease. Pfizer GPS is a one-stop resource for
personalized patient support and specialty pharmacy services for
patients and healthcare professionals. Pfizer GPS employs a dedicated
and caring team of healthcare specialists who are available to help
patients with Gaucher disease and their caregivers with reimbursement
assistance, coordinating and locating infusion services and ongoing
pharmacy support. Using Pfizer GPS, patients and healthcare
professionals can access all support services 24/7 using one toll-free
phone number, 1-855-ELELYSO (1-855-353-5976).
ELELYSO for injection is supplied as 200 units per vial and is available
by prescription only.
For more information about Gaucher disease, please visit www.ELELYSO.com.
ELELYSO™ is indicated for long-term enzyme replacement therapy (ERT) for
adult and pediatric patients with a confirmed diagnosis of Type 1
IMPORTANT SAFETY INFORMATION
Serious hypersensitivity reactions including anaphylaxis have occurred
in some patients treated with ELELYSO (taliglucerase alfa) for
injection, for intravenous use. When treated with ELELYSO your doctor
should monitor you before and after infusion for reactions.
Medical support should be readily available when ELELYSO is given.
Discontinue ELELYSO immediately if you show signs or symptoms of
anaphylaxis during infusion and get immediate medical care. Signs and
symptoms of anaphylaxis included hives, low blood pressure, flushing,
wheezing, chest tightness, nausea, vomiting and dizziness.
Signs and symptoms of hypersensitivity included itching, swelling under
the skin, flushing, redness, rash, nausea, vomiting, cough, chest
tightness and throat irritation. These reactions occurred up to 3 hours
after the start of infusion.
Management of hypersensitivity reactions is based on the severity of the
reaction. Your doctor may manage the reactions by slowing or temporarily
stopping the infusion, and/or treating with medication such as an
antihistamine, a fever reducer and/or corticosteroids for mild
reactions. Treatment with antihistamines and/or corticosteroids prior to
infusion with ELELYSO may prevent these reactions from reoccurring. If
severe hypersensitivity reactions occur, immediately stop the infusion
of ELELYSO and get immediate medical care.
You should be carefully re-evaluated for treatment with ELELYSO if
serious or hypersensitivity reactions including anaphylaxis occur.
The most common adverse reactions for ELELYSO are itching, flushing,
headache, joint pain, pain in extremity, abdominal pain, vomiting,
fatigue, back pain, dizziness, nausea and rash. Vomiting occurred more
often in children than adults.
The recommended dosage of ELELYSO for adults and children who are 4
years of age and older and not taking another ERT is 60 units per kg of
body weight given every other week as a 60 to 120 minute intravenous
As with all therapeutic proteins, including (enzyme replacement therapy)
ERTs, there is a possibility of developing antibodies to ELELYSO. The
relationship between developing antibodies and hypersensitivity
reactions is not clear. Your doctor should monitor you for antibodies to
ELELYSO if you have developed antibodies or if you have experienced
hypersensitivity reactions to ELELYSO or other ERTs.
If you are pregnant, or plan to become pregnant, you should talk to your
doctor about potential benefits and risks.
You are encouraged to report negative side effects of prescription drugs
to the FDA. Visit www.fda.gov/medwatch,
or call 1-800-FDA-1088.
For full prescribing information click here.
The health information contained herein is provided for educational
purposes only and is not intended to replace discussions with a health
care provider. All decisions regarding patient care must be made with a
health care provider, considering the unique characteristics of the
patient. This product information is intended only for residents of the
About Gaucher Disease
Gaucher disease is an inherited lysosomal storage disorder in humans
that affects an estimated 10,000 people worldwide and can cause severe
and debilitating symptoms, including: enlargement of the liver and
spleen, various forms of bone disease, easy bruising, and anemia (a low
number of red blood cells).
Gaucher disease consists of varying degrees of severity; it has been
sub-divided into three subtypes - Types 1, 2, and 3 - according to the
presence or absence of neurological involvement. Type 1, the most
common, is found at a higher frequency among individuals who are of
Ashkenazi Jewish ancestry.
Pfizer and Rare Diseases
Rare diseases are among the most serious of all illnesses and impact
millions of patients worldwide, representing an opportunity to apply our
knowledge and expertise to help make a significant impact in addressing
unmet medical needs. The Pfizer focus on rare diseases builds on more
than a decade of experience and a global portfolio of 22 medicines
approved worldwide that treat rare diseases in the areas of hematology,
neuroscience, inherited metabolic disorders, pulmonology, and oncology.
Pfizer Inc.: Working together for a healthier world®
At Pfizer, we apply science and our global resources to bring therapies
to people that extend and significantly improve their lives. We strive
to set the standard for quality, safety and value in the discovery,
development and manufacture of health care products. Our global
portfolio includes medicines and vaccines as well as many of the world's
best-known consumer health care products. Every day, Pfizer colleagues
work across developed and emerging markets to advance wellness,
prevention, treatments and cures that challenge the most feared diseases
of our time. Consistent with our responsibility as one of the world's
premier innovative biopharmaceutical companies, we collaborate with
health care providers, governments and local communities to support and
expand access to reliable, affordable health care around the world. For
more than 150 years, Pfizer has worked to make a difference for all who
rely on us. To learn more, please visit us at www.pfizer.com.
Protalix BioTherapeutics Inc.
Protalix is a biopharmaceutical company focused on the development and
commercialization of recombinant therapeutic proteins expressed through
its proprietary plant cell-based expression system, ProCellEx®.
Protalix's unique expression system presents a proprietary method for
developing recombinant proteins in a cost-effective, industrial-scale
manner. Protalix's first product manufactured by ProCellEx,
taliglucerase alfa, was approved for marketing by the U.S. Food and Drug
Administration (FDA) in May 2012, by Israel's Ministry of Health in
September 2012, by the Brazilian National Health Surveillance Agency
(ANVISA) in March 2013, by the Mexican Federal Commission for the
Protection against Sanitary Risk (COFEPRIS) in April 2013, by the
Australian Therapeutic Goods Administration (TGA) in May 2014 and by the
regulatory authorities of other countries. Marketing applications for
taliglucerase alfa have been filed in additional territories as well.
Protalix has partnered with Pfizer Inc. for the worldwide development
and commercialization of taliglucerase alfa, excluding Israel and
Brazil, where Protalix retains full rights. Protalix's development
pipeline includes the following product candidates: PRX-102, a modified
version of the recombinant human alpha-GAL-A protein for the treatment
of Fabry disease; PRX-112, an orally-delivered glucocerebrosidase enzyme
that is produced and encapsulated within carrot cells, for the treatment
of Gaucher disease; pr-antiTNF, a similar plant cell version of
etanercept (Enbrel®) for the treatment of certain immune and
inflammatory diseases, such as rheumatoid arthritis, Crohn's disease,
colitis, psoriasis and other autoimmune and inflammatory disorders;
PRX-110 for the treatment of Cystic Fibrosis; and others.
Protalix Forward Looking Statement Disclaimer
To the extent that statements in this press release are not strictly
historical, all such statements are forward-looking, and are made
pursuant to the safe-harbor provisions of the Private Securities
Litigation Reform Act of 1995. The terms "anticipate," "believe,"
"estimate," "expect," "plan" and "intend" and other words or phrases of
similar import are intended to identify forward-looking statements. Drug
discovery and development involve a high degree of risk. Factors that
might cause material differences include, among others: failure or delay
in the commencement or completion of our preclinical studies and
clinical trials which may be caused by several factors, including: risks
related to the commercialization efforts for taliglucerase alfa in the
United States, Israel, Brazil and other countries in which it is
approved for sale; risks relating to the review process of other foreign
regulatory and other governmental bodies; risks relating to delays in
other foreign regulatory authorities' approval of any applications filed
for taliglucerase alfa or refusals to approve such filings, as well as
the decisions of such regulatory authorities regarding labeling and
other matters that could affect the availability of taliglucerase alfa
or its commercial potential; the risk that applicable regulatory
authorities may refuse to approve the marketing and sale of a drug
product even after acceptance of an application filed for the drug
product; the dependence on performance by third party providers of
services and supplies relating to the commercialization of taliglucerase
alfa; the inherent risks and uncertainties in developing drug platforms
and products of the type we are developing; the impact of development of
competing therapies and/or technologies by other companies and
institutions; potential product liability risks, and risks of securing
adequate levels of product liability and other necessary insurance
coverage; and other factors described in our filings with the U.S.
Securities and Exchange Commission. These forward-looking statements are
based on current information that may change and you are cautioned not
to place undue reliance on these forward-looking statements. The
statements in this release are valid only as of the date hereof and we
disclaim any obligation to update this information. All
forward-looking statements are qualified in their entirety by this
Pfizer Disclosure Notice
The information contained in this release is as of August 28, 2014.
Pfizer assumes no obligation to update forward-looking statements
contained in this release as the result of new information or future
events or developments.
This release contains forward-looking information about ELELYSO ™
(taliglucerase alfa) and about an indication in the U.S. for ELELYSO for
the long-term treatment of pediatric patients with a confirmed diagnosis
of Type 1 Gaucher disease, including their potential benefits, that
involves substantial risks and uncertainties that could cause actual
results to differ materially from those expressed or implied by such
statements. Risks and uncertainties include, among other things, the
uncertainties regarding the commercial success of ELELYSO and the
indication for pediatric use in the U.S.; whether and when applications
will be filed for ELELYSO in other jurisdictions and whether and when
regulatory authorities in other jurisdictions will approve applications
for ELELYSO, as well as their decisions regarding labeling and other
matters that could affect the availability or commercial potential of
ELELYSO; and competitive developments.
A further description of risks and uncertainties can be found in
Pfizer's Annual Report on Form 10-K for the fiscal year ended December
31, 2013 and in its subsequent reports on Form 10-Q, including in the
sections thereof captioned "Risk Factors" and "Forward-Looking
Information That May Affect Future Results", as well as in its
subsequent reports on Form 8-K, all of which are filed with the SEC (News - Alert) and
available at www.sec.gov
© 2014 Pfizer Inc. All rights reserved. TAL685104
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