European Medicines Agency's COMP Adopts Positive Opinion for the Orphan Drug Designation for Protalix's taliglucerase alfa
January 13 2010 - 7:30AM
PR Newswire (US)
CARMIEL, Israel, Jan. 13 /PRNewswire/ -- Protalix BioTherapeutics,
Inc. (NYSE-Amex: PLX), announced today that the Committee for
Orphan Medicinal Products (COMP) of the European Medicines Agency
(EMEA), after reviewing all relevant clinical data, has recommended
that the European Commission grant orphan drug designation to
taliglucerase alfa, the Company's proprietary plant cell expressed
recombinant form of glucocerebrosidase for the treatment of Gaucher
disease. The U.S. Food and Drug Administration (FDA) granted orphan
drug designation and fast track designation to taliglucerase alfa
in 2009. Orphan drug designation in Europe is granted to medicinal
products intended for the diagnosis, prevention and treatment of
life-threatening diseases and very serious conditions that affect
not more than five in 10,000 people in the European Union. Orphan
drug designation is generally given to medicinal products that
treat conditions for which no current therapy exists or are
expected to bring a significant benefit to patients over existing
therapies. If granted by the European Commission, orphan drug
designation will provide the Company with a centralized procedure
for obtaining marketing authorization for taliglucerase alfa, with
a single marketing authorization valid throughout all EU Member
States. The Company may also be eligible for a number of additional
incentives including protocol assistance, reduction in registration
fees and eligibility for grants and initiatives supporting research
and development related to the orphan drug designation. "This is an
important step in our global commercialization strategy for
taliglucerase alfa," said Dr. Einat Brill Almon, the Company's
Senior Vice President, Product Development. "We plan to file a
marketing authorization application with the European Medicines
Agency for taliglucerase alfa in the upcoming months." The Company
reported positive top-line results from its pivotal phase III
clinical trial of taliglucerase alfa in October 2009. Full results
from this study will be presented at the Annual Meeting of the
Lysosomal Disease Network: WORLD Symposium 2010, February 10-12, in
Miami, Florida. In addition, the Company completed the filing of a
New Drug Application for taliglucerase alfa with the FDA in
December 2009. In November 2009, the Company entered into a license
and supply agreement with Pfizer Inc. (NYSE:PFE) pursuant to which
the Company granted Pfizer the exclusive worldwide rights to
develop and commercialize taliglucerase alfa for the treatment of
Gaucher disease, except for Israel. The Company retained the right
to develop and commercialize taliglucerase alfa in Israel. About
Gaucher disease Gaucher disease, an inherited condition, is the
most prevalent lysosomal storage disorder, with an incidence of
about 1 in 20,000 live births. People with Gaucher disease do not
have enough of an enzyme, beta-glucosidase (glucocerebrosidase)
that breaks down a certain type of fat molecule. As a result, lipid
engorged cells (called Gaucher cells) amass in different parts of
the body, primarily the spleen, liver and bone marrow. Accumulation
of Gaucher cells may cause spleen and liver enlargement, anemia,
excessive bleeding and bruising, bone disease and a number of other
signs and symptoms. About Protalix Protalix is a biopharmaceutical
company focused on the development and commercialization of
proprietary recombinant therapeutic proteins expressed through its
proprietary plant cell based expression system. Protalix's
ProCellEx(TM) presents a proprietary method for the expression of
recombinant proteins that Protalix believes will allow for the
cost-effective, industrial-scale production of recombinant
therapeutic proteins in an environment free of mammalian components
and viruses. Protalix is also advancing additional recombinant
biopharmaceutical drug development programs. Taliglucerase alfa is
an enzyme replacement therapy in development under a Special
Protocol Assessment with the FDA for Gaucher disease. Safe Harbor
Statement: 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. 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 relating to: the successful preclinical development
of our product candidates; the completion of clinical trials; the
review process of the FDA, the EMEA, other foreign regulatory
bodies and other governmental regulatory bodies, including the
FDA's and the EMEA's review of any filings we make in connection
with the treatment protocol; delays in the FDA's, the EMEA's or
other health regulatory authorities' approval of any applications
we file or refusals to approve such filings; refusals by such
regulatory authorities to approve the marketing and sale of a drug
product even after acceptance of an application we file for any
such drug product; the identification of lead compounds; the risk
that we may fail to satisfy certain conditions relating to grants
we have received from the Office of the Chief Scientist of Israel's
Ministry of Industry and Trade which may lead to our being required
to refund grants previously received together with interest and
penalties; the risk that the Office of the Chief Scientist may not
deliver to us all of the funds awarded to us; uncertainties related
to the ability to attract and retain partners for our technologies
and products under development; and other factors described in our
filings with the Securities and Exchange Commission. Companies in
the pharmaceutical and biotechnology industries have suffered
significant setbacks in advanced or late-stage clinical trials,
even after obtaining promising earlier trial results or in
preliminary findings for such clinical trials. Further, even if
favorable testing data is generated by clinical trials of drug
products, the FDA, EMEA or any other foreign regulatory authority
may not accept or approve an NDA filed by a pharmaceutical or
biotechnology company for such drug product. Failure to obtain
approval from the FDA, EMEA or any other foreign regulatory
authority of any of our drug candidates in a timely manner, if at
all, will severely undermine our business and results of operation
by reducing our potential marketable products and our ability to
generate corresponding product revenues. The statements in this
release are valid only as of the date hereof and we disclaim any
obligation to update this information. Contact: Marcy Nanus The
Trout Group, LLC Telephone: 646-378-2927 Email: Media Contact: Brad
Miles BMC Communications Group, LLC Telephone: 212-477-9007 x17
Email: DATASOURCE: Protalix BioTherapeutics, Inc. CONTACT: Marcy
Nanus of The Trout Group, LLC, +1-646-378-2927, ; or Media: Brad
Miles of BMC Communications Group, LLC, +1-212-477-9007 x17, , both
for Protalix BioTherapeutics, Inc.
Copyright