Protalix : FDA approuve le "treatment protocol" pour le prGCD
Subject: Protalix - press
release: U.S. Food and Drug Administration Approves Protalix's Treatment
Protocol for prGCD
CARMIEL, Israel--(BUSINESS WIRE)--Protalix BioTherapeutics, Inc.
(NYSE-Amex:PLX), announced today that the U.S. Food and Drug Administration
(FDA) has approved the Company’s treatment protocol for prGCD, the Company’s
proprietary plant-cell expressed recombinant form of glucocerebrosidase (GCD)
for the treatment of Gaucher disease. The treatment protocol allows physicians
and other care-providers to treat patients of Gaucher disease with prGCD in the
United States and additional countries world-wide while studies of prGCD
continue as part of the Company’s ongoing pivotal Phase III clinical trial.
Prior to accepting the protocol, the FDA reviewed available data from the
Company’s on-going Phase III clinical development programs.
The treatment protocol is a multicenter, open-label trial designed to
allow physicians and other care-providers to treat patients of Gaucher disease
with prGCD during the expected shortage of Cerezyme® and thereafter. Cerezyme®
is a mammalian cell expressed version of glucocerebrosidase and the only enzyme
replacement therapy currently approved for Gaucher disease. The treatment
protocol allows patients enrolled in the protocol to continue being treated
with prGCD until its anticipated marketing approval from the FDA. The Company
will provide the drug free of charge to patients enrolled in the protocol.
"We appreciate the guidance and vote of confidence provided by the
FDA in establishing a treatment protocol for prGCD and are working closely with
physicians and patient advocacy groups to allow Gaucher disease patients to
gain access to our drug," commented Dr. David Aviezer, the Company’s
President and Chief Executive Officer. "We expect to conclude our phase
III pivotal study next month and are looking forward to announcing top-line
results from this study in October. We anticipate filing an NDA with the FDA by
the end of this year.”
About Protalix BioTherapeutics
Protalix is a biopharmaceutical company. Its goal is to become a fully
integrated biopharmaceutical company focused on the development and
commercialization of proprietary recombinant therapeutic proteins to be
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 conducting a
Phase III pivotal study for its lead product candidate, prGCD, to be used in
enzyme replacement therapy for Gaucher disease, a rare and serious lysosomal
storage disorder in humans with severe and debilitating symptoms. Protalix and
the U.S. Food and Drug Administration agreed on the final design of the pivotal
Phase III clinical trial through the FDA’s Special Protocol Assessment (SPA)
process. Protalix has completed enrollment for this study and is treating
patients in the study in North America, South America, Israel, Europe and South
Africa. The study is monitored by an independent Data Monitoring Committee,
including experts in the field, who monitor the on-going safety data, which has
recently held their last scheduled meeting before the end of the trial. No
serious adverse events have been reported in the study. Protalix is also
advancing additional recombinant biopharmaceutical drug development programs