Pfizer Inc.(NYSE:PFE) and Protalix BioTherapeutics, Inc. (NYSE-AMEX:PLX,
TASE:PLX) announced today that the United States (U.S.) Food and Drug
Administration (FDA) approved ELELYSO™ (taliglucerase alfa) for
injection, an enzyme replacement therapy (ERT) for the long-term
treatment of adults with a confirmed diagnosis of type 1 Gaucher disease.
ELELYSO is the first FDA-approved plant cell-based ERT for Gaucher
disease. It is also the first approved plant cell-expressed drug that is
derived from ProCellEx®, Protalix's proprietary manufacturing
system, using genetically engineered carrot cells. ELELYSO™ is a form of
the human lysosomal enzyme, glucocerebrosidase, used to treat Gaucher
"The approval of ELELYSO is important for patients who
depend on available ERT to manage their Gaucher disease. At Protalix,
our passion to develop ELELYSO was strongly driven by our personal
experience with family members and friends who have to live with this
disease every day," said David Aviezer, Ph.D., MBA, president and chief
executive officer of Protalix BioTherapeutics. "We also believe that
this great news is a recognition of our technology, which is a plant
cell manufacturing system from Protalix. This technology is the
production process behind ELELYSO and other Protalix product candidates."
Supply disruptions of approved ERTs have been affecting those living
with Gaucher disease since 2009 in multiple countries including the U.S.
To help minimize the possibility of supply disruptions, Pfizer is
launching the "Supply Continuity Program," which will endeavor to
maintain a continuously restocked 24 months of supply at various stages
of production for U.S. patients prescribed ELELYSO.
With the approval of ELELYSO, Pfizer is also introducing Gaucher
Personal Support (GPS), a specialized support program for people living
with Gaucher disease. Through the GPS program, Pfizer will provide an
assistance program covering 100 percent of prescription co-pay expenses
for eligible patients on ELELYSO who have commercial health insurance.
Additionally, Pfizer will provide financial assistance for eligible
patients who are uninsured or under-insured where allowed by law.
GPS is a one-stop resource for patient support and specialty pharmacy
services for those taking ELELYSO. GPS staffs a dedicated team of health
care specialists who are available 24/7 to help Gaucher disease patients
and their families with reimbursement assistance, facilitate locating
infusion services, and provide ongoing pharmacy support. Patients can
call 1-855-ELELYSO (1-855-353-5976) for a free patient information kit
about ELELYSO and the GPS program.
"With ELELYSO, Pfizer is proud to be making a difference in the lives of
people living with Gaucher disease," said Diem Nguyen, Ph.D., MBA,
general manager, Pfizer Biosimilars. "ELELYSO is an important addition
to Pfizer's rare disease portfolio."
There are currently two other ERTs approved for Gaucher disease in the
U.S., imiglucerase and velaglucerase. Taliglucerase alfa for injection
(ELELYSO) will be available to U.S. patients at a cost that will
be priced at 25 percent below the cost of imiglucerase.
"I am delighted that the Gaucher community will have another treatment
option. It is especially important for this group of patients who have
suffered from supply shortages in recent years," said Rhonda Buyers, CEO
and executive director of the National Gaucher Foundation (NGF).