Full Press Release Details
UPLYSO (alfataliglicerase) Approved
for the Treatment of Gaucher Disease
CARMIEL, Israel, April 29, 2013 /GlobeNewswire
/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
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
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
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