Protalix BioTherapeutics Receives Marketing Authorization for Elelyso(TM) for the Treatment of Gaucher Disease From the Israeli
September 27 2012 - 6:30AM
Protalix BioTherapeutics, Inc. (NYSE-MKT:PLX) (TASE:PLX), announced
today that is has received marketing authorization from the Israeli
Ministry of Health for ElelysoTM (taliglucerase alfa) for
injection, an enzyme replacement therapy (ERT) for the long-term
treatment of adults with Type 1 Gaucher disease. Elelyso will be
marketed in Israel by Protalix Ltd., the holder of all marketing
rights to Elelyso in the Israeli market.
This is the second marketing approval of Elelyso, which was
approved by the U.S. Food and Drug Administration (FDA) on May 1,
2012. Marketing applications have been filed in additional
territories. Elelyso is marketed in the United States by the
Company's commercialization partner, Pfizer Inc. ("Pfizer").
Under its development and commercialization arrangement with
Pfizer, the Company maintained the commercialization rights to
Elelyso in Israel. Accordingly, the Company has built an
internal marketing team designed to serve the Israeli
market. The Company intends to sell Elelyso in Israel at a
competitive price compared to other products already available to
Gaucher patients. Over the past five years, the Company has
treated over 60 Gaucher patients in Israel with Elelyso through
clinical trials and compassionate use programs and expects that a
substantial proportion of these patients will soon be treated
through commercial programs.
Elelyso is the first plant cell-based biopharmaceutical approved
for marketing by the Israeli Ministry of Health. It is also
the first plant cell-expressed drug derived from ProCellEx® to
achieve regulatory approval for marketing. ProCellEx is the
Company's proprietary plant cell-based protein expression
system. Elelyso is a form of the human lysosomal enzyme,
glucocerebrosidase, used to treat Gaucher disease.
"We are very excited to have our first drug product approved in
our home country," said Dr. David Aviezer, President and Chief
Executive Officer of Protalix BioTherapeutics. "In our
development efforts, we enjoyed the cooperation of the leading
Israeli medical and academic institutions, and we rely in part on
support from research grants from the Israeli government. We
are proud that our Gaucher disease treatment will be available for
commercial sale in Israel."
The Israeli Ministry of Health's marketing authorization of
Elelyso was based on its review of data compiled by the Company
from its pivotal phase III clinical trial, as well as data from its
extension trial in which treatment-naïve patients that were treated
with taliglucerase alfa for a 24-month period, and from the
Company's switchover trial which collected data from Gaucher
patients that had previously been treated with imiglucerase
(Cerezyme®) and were switched to treatment with taliglucerase alfa
(Elelyso).
"The Israeli approval of Elelyso is important for local Gaucher
patients," said Mr. Yossi Cohen, Chairman of the Israeli
Association for Gaucher. "Given the inconsistent supply of ERT
for the treatment of Gaucher disease worldwide in recent years, we
believe the addition of a new treatment for Gaucher patients will
provide them with greater confidence regarding treatment. The
fact that the product is manufactured locally by an Israeli company
increases our excitement about this approval."
"The clinical studies of Elelyso to date, both the pivotal and
the extension studies, demonstrate that Elelyso is an effective
treatment for Gaucher disease," said Professor Ari Zimran, M.D.,
Director of the Gaucher Clinic, Shaare Zedek Medical Center,
Jerusalem, Israel. "The results of the Company's 24-month
naive extension trial and switch over study support Elelyso as an
important treatment alternative for Gaucher patients in
Israel."
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 Elylyso (taliglucerase alfa)
Elylyso (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,
ELELYSO™ (taliglucerase alfa), was approved for marketing by the
U.S. Food and Drug Administration on May 1, 2012. Protalix is
partnered with Pfizer Inc. for worldwide development and
commercialization, excluding Israel, where Protalix retains full
rights. Marketing applications for taliglucerase alfa have
been filed in additional territories as well. 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 Israel; 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; our dependence on performance by third party
providers of services and supplies relating to the
commercialization of taliglucerase alfa in Israel; 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
Tricia Swanson
The Trout Group, LLC
646-378-2953
tswanson@troutgroup.com
Media Contact
Jennifer Conrad or Kari Watson
MacDougall Biomedical Communications
781-235-3060
jconrad@macbiocom.com
kwatson@macbiocom.com