Pfizer Inc. (NYSE:PFE) and Protalix BioTherapeutics, Inc. (NYSE
MKT:PLX) (TASE:PLX) announced today that the Brazilian National
Health Surveillance Agency (ANVISA, Agencia Nacional de Vigilancia
Sanitaria) has granted regulatory approval to UPLYSO™
(alfataliglicerase) for the long-term enzyme replacement therapy
for adults with a confirmed diagnosis of Type I Gaucher disease.
Gaucher disease is a rare lysosomal storage disorder that affects
approximately 10,000 people worldwide.
UPLYSO is known as ELELYSO™ (taliglucerase alfa) outside of
Latin America. ELELYSO was approved by the United States Food and
Drug Administration in May 2012 and by Israel's Ministry of Health
in September 2012 for the long-term enzyme replacement therapy
(ERT) for adults with a confirmed diagnosis of Type I Gaucher
disease. The ANVISA approval comes after the approval of UPLYSO by
the Ministry of Public Health in Uruguay in November 2012.
"ANVISA's approval of UPLYSO demonstrates Pfizer's ability to
successfully bring therapies for rare diseases to the marketplace,"
said Diem Nguyen, Ph.D., MBA, general manager, Pfizer Biosimilars.
"We are committed to the Gaucher community and look forward to
continuing to work closely with our partner Protalix toward our
goal of bringing UPLYSO to those living with Gaucher throughout
Latin America."
UPLYSO is the first plant cell-based ERT for the treatment of
Gaucher disease. It is also the first approved plant cell-expressed
drug that is derived from ProCellEx®, Protalix's proprietary plant
cell-based protein manufacturing system, using genetically
engineered carrot cells. UPLYSO is a form of the human lysosomal
enzyme, glucocerebrosidase, used to treat Gaucher disease.
"The approval of UPLYSO provides an important treatment option
for those in Brazil using enzyme replacement therapy to manage
their Gaucher disease and demonstrates the potential of our plant
cell manufacturing technology," said David Aviezer, Ph.D., MBA,
president and chief executive officer of Protalix
BioTherapeutics.
On November 30, 2009, Protalix entered into an agreement with
Pfizer to develop and commercialize taliglucerase
alfa/alfataliglicerase. Under the terms of the agreement, Protalix
retained the exclusive commercialization rights in Israel, while
Pfizer received exclusive licensing rights for commercialization in
all other markets.
Indication for UPLYSO in Brazil
UPLYSO (alfataliglicerase) is indicated for long-term enzyme
replacement therapy for adults with a confirmed diagnosis of Type I
Gaucher disease. The manifestations of Gaucher disease may include
one or more of the following: splenomegaly, hepatomegaly, anemia,
thrombocytopenia, bone disease.
Important Safety Information for UPLYSO in
Brazil
As with any intravenous protein medicine, like enzyme
replacement therapy (ERT), UPLYSO may cause infusion-related
reactions (i.e., occurring during or shortly after infusion) and
hypersensitivity reactions. If a severe allergic reaction occurs,
immediate discontinuation of the alfataliglicerase infusion is
recommended. Patients who experience reactions of hypersensitivity
or related to the infusion, can however be managed successfully.
The therapy can be continued by slowing the infusion rate, treating
with medicinal products such as antihistamines, antipyretics and/or
corticosteroids, and/or stopping and resuming treatment with
decreased infusion rate. Pre-treatment with antihistamines and/or
corticosteroids may prevent subsequent reactions.
The most commonly observed infusion reactions were headache,
hypersensitivity (allergic reaction), dizziness, flushing
(redness), throat irritation, nausea (qualm), pruritus (itch),
erythema (redness), rash, bone pain, back pain, arthralgia (joint
pain), among others.
Also, there is a possibility of developing antibodies to UPLYSO.
However, it is currently unclear whether this has an impact on the
clinical response or adverse reactions. Patients with an immune
response to other ERTs who are switching to UPLYSO should continue
to be monitored for antibodies. Patients who have developed
infusion or immune reactions with UPLYSO or with another ERT should
be monitored for antidrug antibodies when being treated with
UPLYSO.
If you are pregnant, or plan to become pregnant, you should talk
to your doctor about potential benefits and risks.
Indication for ELELYSO in the U.S.
ELELYSO™ (taliglucerase alfa) for injection is a hydrolytic
lysosomal glucocerebroside-specific enzyme indicated for long-term
enzyme replacement therapy (ERT) for adults with a confirmed
diagnosis of Type 1 Gaucher disease.
Important Safety Information for ELELYSO in the
U.S.
As with any intravenous protein medicine, like enzyme
replacement therapy (ERT), severe allergic reactions (including
anaphylaxis) have been observed in patients treated with ELELYSO.
If this occurs, ELELYSO should be immediately discontinued, and
appropriate medical treatment should be initiated. Patients who
have experienced anaphylaxis to ELELYSO or another ERT should
proceed with caution upon retreatment.
In addition, infusion reactions (including allergic
reactions)—defined as a reaction occurring within 24 hours of the
infusion—were the most commonly observed reactions to ELELYSO. The
most commonly observed infusion reactions were headache, chest pain
or discomfort, weakness, fatigue, hives, abnormal redness of the
skin, increased blood pressure, back or joint pain, and flushing.
Other infusion or allergic reactions included swelling of the face,
mouth, and/or throat; wheezing; shortness of breath; skin color
turning blue; coughing; and low blood pressure. Most of these
reactions were mild and did not require treatment.
Management of infusion reactions is based on the type and
severity of the reaction. Your doctor may manage infusion reactions
by temporarily stopping the infusion, slowing the infusion rate, or
treating with medications such as an antihistamine and/or a fever
reducer. Treatment with antihistamines and/or corticosteroids prior
to infusion with ELELYSO may prevent these reactions.
Other common adverse reactions observed were upper respiratory
tract infections, throat infection, flu, urinary tract infection,
and pain in extremities.
As with all therapeutic proteins, including ERTs, there is a
possibility of developing antibodies to ELELYSO. However, it is
currently unclear whether this has an impact on the clinical
response or adverse reactions. Patients with an immune response to
other ERTs who are switching to ELELYSO should continue to be
monitored for antibodies. Comparison of the frequency of antibodies
across ERTs may be misleading. Patients who have developed infusion
or immune reactions with ELELYSO or with another ERT should be
monitored for antidrug antibodies when being treated with
ELELYSO.
If you are pregnant, or plan to become pregnant, you should talk
to your doctor about potential benefits and risks.
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
United States.
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.
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.
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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 protein
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, ELELYSO™ (taliglucerase alfa),
was approved for marketing by the U.S. Food and Drug Administration
on May 1, 2012, by Israel's Ministry of Health in September 2012
and by ANVISA on March 18, 2013. It also has been approved in
Uruguay. Protalix has partnered with Pfizer Inc. for the
worldwide development and commercialization of ELELYSO™, excluding
Israel, where Protalix retains full rights. Marketing
applications for taliglucerase alfa have been filed in additional
markets.
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. Forward-looking
statements involve substantial risks and uncertainties. Such risks
and uncertainties include, among other things, the uncertainties
related to the timing of a commercial launch in Brazil and other
countries; decisions by regulatory authorities in various countries
regarding whether and when to approve drug applications that have
been or may be filed for taliglucerase alfa in such countries as
well as their decisions regarding labeling and other matters that
could affect its availability or 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; and risks related to
competitive developments. The statements in this release are valid
only as of the date hereof, and Protalix disclaims any obligation
to update this information. These and other risks and uncertainties
are detailed under the heading "Risk Factors" in Protalix's Annual
Report on Form 10-K for the year ended December 31, 2012.
PFIZER DISCLOSURE NOTICE: The information contained in this
release is as of March 18, 2013. 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 statements about
taliglucerase alfa (generic name: alfataliglicerase in Brazil,
trade name: UPLYSO in Brazil) that involve substantial risks and
uncertainties. Such risks and uncertainties include, among other
things, the uncertainties regarding the commercial success of
UPLYSO in Brazil; decisions by regulatory authorities in various
other countries regarding whether and when to approve drug
applications that have been or may be filed for taliglucerase alfa
in such countries as well as their decisions regarding labeling and
other matters that could affect its availability or commercial
potential; 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, 2012 and in its reports on Form 10-Q and Form 8-K.
CONTACT: Pfizer Contacts:
Sharon Castillo (Media)
202-494-6769
Sharon.J.Castillo@pfizer.com
Suzanne Harnett (Investors)
212-733-8009
Suzanne.Harnett@pfizer.com
Protalix Contacts:
Kari Watson (Media)
MacDougall Biomedical
Communications
781-235-3060
kwatson@macbiocom.com
Marcy Beth Nanus (Investors)
The Trout Group, LLC
646-378-2927
MNanus@troutgroup.com