Protalix BioTherapeutics, Inc. (NYSE-MKT:PLX) (TASE:PLX), announced
today that the Mexican Federal Commission for the Protection
against Sanitary Risk (COFEPRIS) and the Public Health Institute of
Chile have both granted regulatory approval to UPLYSO™
(alfataliglicerase) for the long-term enzyme replacement therapy
for adults with a confirmed diagnosis of Type I Gaucher disease.
UPLYSO will be marketed in Mexico and Chile by Pfizer Inc., the
Company's commercialization partner.
UPLYSO is known as ELELYSO™ (taliglucerase alfa) outside of
Latin America. Taliglucerase alfa was approved by the U.S. Food and
Drug Administration in May 2012 and subsequently has been approved
by Israel's Ministry of Health, Brazil's ANVISA and by the
regulatory authorities of other countries.
"We are very happy that UPLYSO may now be made available as an
additional treatment alternative to Gaucher patients in Mexico and
Chile," stated Dr. Raul Chertkoff, the Company's Vice President,
Medical Affairs. "Including Mexico and Chile, UPLYSO has been
reviewed and approved by six different regulatory authorities
across the globe. Through our joint efforts with Pfizer, we
are working on expanding the commercial potential for UPLYSO in
additional countries."
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®, the Company'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.
On November 30, 2009, the Company entered into an agreement with
Pfizer to develop and commercialize taliglucerase
alfa/alfataliglicerase. Under the terms of the agreement, the
Company retained the exclusive commercialization rights in Israel,
while Pfizer received exclusive licensing rights for
commercialization in all other markets.
Indication for ELELYSO in the United States
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
United States
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.
About ELELYSO (taliglucerase alfa)
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.
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 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, where Protalix retains full
rights. Protalix's development pipeline also 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-105, a pegylated recombinant human
acetylcholinesterase in development for several therapeutic and
prophylactic indications, a biodefense program and an
organophosphate-based pesticide treatment program; an
orally-delivered glucocerebrosidase enzyme that is naturally
encased in carrot cells, also for the treatment of Gaucher disease;
pr-antiTNF, a similar plant cell version of etanercept (Enbrel®)
for the treatment of certain immune diseases such as rheumatoid
arthritis, juvenile idiopathic arthritis, ankylosing spondylitis,
psoriatic arthritis and plaque psoriasis; and others.
Forward Looking Statements
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. These forward-looking
statements are subject to known and unknown risks and uncertainties
that may cause actual future experience and results to differ
materially from the statements made. These statements are
based on our current beliefs and expectations as to such future
outcomes. Drug discovery and development involve a high degree
of risk. Factors that might cause material differences
include, among others: risks and uncertainties related to the
timing of a commercial launch and market acceptance of
taliglucerase alfa in Mexico and Chile; 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; 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 in Mexico and Chile; 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. The statements in
this release are valid only as of the date hereof and we disclaim
any obligation to update this information.
CONTACT: Investor Contact
Marcy Nanus
The Trout Group, LLC
646-378-2927
mnanus@troutgroup.com
Media Contact
Jennifer Conrad or Kari Watson
MacDougall Biomedical Communications
781-235-3060
jconrad@macbiocom.com
kwatson@macbiocom.com