ISTH Symposia Discuss Prevention and Eradication of FVIII Inhibitors and
Introduce First Recombinant FVIII Produced From a Human Cell Line
LACHEN, Switzerland and HOBOKEN, N.J., Aug. 26 /CNW/ -- 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
"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
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 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 (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, please
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