MEDIA RELEASE PR35829
Octapharma Targeting the Major Risk of Hemophilia Treatment - FVIII Antibodies
LACHEN and HOBOKEN, N.J., Aug. 26 /PRNewswire-AsiaNet/ --
ISTH Symposia Discuss Prevention and Eradication of FVIII Inhibitors and
Introduce First Recombinant FVIII Produced From a Human Cell Line
Octapharma AG is leading an international initiative focused on confronting the major risk associated with
hemophilia A therapy - anti-factor VIII (FVIII) antibodies, also known as inhibitors. This initiative, combined with
Octapharma's efforts to pursue the first recombinant FVIII therapy produced from a human cell line, could
dramatically impact the treatment of an estimated one in every 5,000 to 10,000 men born with hemophilia A
worldwide. Globally, 75% of the hemophilia cases go undiagnosed or untreated.
Octapharma AG, the third largest plasma products manufacturer in the world, recently brought together
many of the most respected blood coagulation disorder researchers for two symposia on this important issue
during the International Society on Thrombosis and Haemostasis (ISTH) Biennial Congress in Boston.
Octapharma was one of only five Platinum Sponsors at the ISTH Congress, which attracted approximately
7,500 representatives of the medical and research community as well as many patients.
Clinical experts noted during the symposia that up to 40 percent of previously untreated patients (PUPs)
with hemophilia A develop the most serious clinical complication of FVIII replacement therapy - inhibitory
antibodies - which can result in uncontrolled hemorrhage, increased hospitalizations and joint damage,
resulting in increased morbidity and mortality.
"We brought the international community together to confront this issue because our worldwide commitment
to hemophilia A patients dates back to Octapharma's formation 25 years ago," said Octapharma AG Vice
Chairman Kim Bjornstrup. "Our first therapies were developed for the hemophilia community and today most
hemophilia patient advocates say that inhibitor development is the greatest obstacle to effective treatment.
Octapharma is focused on introducing innovative development strategies that will help improve patient quality
of life by finding an effective treatment and developing products to overcome this obstacle."
The Octapharma-sponsored symposium "Prevention and Eradication of FVIII Inhibitors: Bridging Lab and
Field Research" was chaired by David Lillicrap, M.D., Professor of Pathology and Molecular Medicine at
Queen's University in Ontario, Canada, and Georges E. Rivard, M.D., Professor of Pediatrics at Universite de
Montreal. The symposium provided an opportunity for discussing recent data supporting the use of von
Willebrand factor (VWF)/FVIII concentrates for Immune Tolerance Induction (ITI) in hemophilia A patients with
poor prognosis for a successful ITI outcome.
Presenters reviewed a balanced mix of clinical, preclinical and basic data on the role of the presence of
VWF in FVIII concentrates and the clinical experience on prevention and eradication of FVIII inhibitors. In
addition, the most recent prospective and retrospective clinical data obtained with different classes of FVIII
concentrates used for ITI treatment were presented along with an update on the progress of the ongoing
prospective study designed to further investigate the risk of FVIII inhibitor development in previously untreated
patients with hemophilia.
"A growing body of clinical experience suggests that VWF-containing pdFVIII concentrates increase the
likelihood of successful tolerization, particularly in patients with poor prognostic factors," said symposium
presenter Wolfhart Kreuz, M.D. of the Hemophilia Comprehensive Care Centre at Johann Wolfgang Goethe
University Hospital in Frankfurt, Germany. "Successful ITI leads to normalization of the FVIII half-life, allows
fully effective on-demand replacement therapy and prophylaxis, with consequent improvement in the patient's
quality of life and a marked reduction in the cost of treatment."
The Octapharma-sponsored symposium "From Humans to Humans - Introducing the First Recombinant
FVIII Produced From a Human Cell Line" was chaired by Edward G.D. Tuddenham, M.D., Director of The
Katharine Dormandy Haemophilia Center & Thrombosis Unit in London, and Johannes Oldenburg, M.D.,
Ph.D., Chairman and Director of the Institute of Experimental Haematology and Transfusion Medicine in Bonn,
Germany.
"Twenty years after the start of clinical trials with rFVIII concentrates expressed by hamster cells, a new
rFVIII compound has recently entered clinical studies," Dr. Tuddenham said. "The primary goal behind the
development of this new rFVIII was to reduce the overall immunogenic challenge (and resultant inhibitor
formation) to the hemophilia patient during rFVIII replacement therapy. An essential part of this strategy was
the development of a human rFVIII protein expressed in a human cell-based protein expression system
instead of using existing hamster-derived cell lines."
The symposium highlighted the benefits of using a human cell-based protein; preclinical characterizations
and some of the functional properties of the first recombinant factor VIII (rFVIII) from a human cell-line; and the
planned global clinical development program with the new rFVIII derived from human cells. The investigational
new drug application for Octapharma's human cell line rFVIII was filed in the U.S. in May 2008. Clinical trials
started in Russia in Spring 2009 and are expected to start in the U.S. later this year. Additionally, Octapharma
has other recombinant human cell line products in various stages of development.
During the symposium, possible advantages of using a human cell line for recombinant FVIII production
were presented. Those include the absence of the rodent antigenic epitopes Gal-1,3-Galactose and N-
glycolyl-neuraminic-acid on the FVIII-protein, which could lead to a reduced immunogenicity compared to
currently marketed recombinant factor VIII products that are all derived from hamster cell lines and also to a
potentially increased tolerability.
Octapharma AG
Octapharma AG is headquartered in Lachen, Switzerland, the third largest plasma products manufacturer in
the world and has been committed to patient care and medical innovation for over 25 years. Octapharma's
core business is the development, production and sale of high quality human protein therapies from both
human plasma and human cell lines, including immune globulin intravenous (IGIV). In the U.S., Octapharma's
IGIV product, octagam(R) (immune globulin intravenous [human] 5%), is used to treat disorders of the immune
system, and Octapharma's Albumin (Human) is indicated for the restoration and maintenance of circulating
blood volume. Octapharma employs over 3,000 people and has biopharmaceutical experience in 80 countries
worldwide, including the United States, where Octapharma USA is headquartered in Hoboken, N.J.
Octapharma operates two state-of-the-art production sites licensed by the U.S. Food and Drug Administration,
providing the highest level of production flexibility and minimizing product shortages. For more information,
Forward-looking statements
This news release contains forward-looking statements, which include known and unknown risks,
uncertainties and other factors not under the company's control. The company assumes no liability whatsoever
to update these forward-looking statements or to conform them to future events or developments. These
factors include results of current or pending research and development activities and actions by the FDA or
other regulatory authorities.
SOURCE: Octapharma AG
CONTACT: Fred Feiner of Yankee Public Relations,
+1-908-894-3930,
fred@yankeepr.com,
for Octapharma AG
NEWS 
