LACHEN, Switzerland and HOBOKEN, N.J., Aug. 26 /PRNewswire/ -- 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.
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.
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.
"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."
"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."
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.
Forward-looking statements
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SOURCE Octapharma AG
CONTACT: Fred Feiner of Yankee Public Relations, +1-908-894-3930,
fred@yankeepr.com, for Octapharma AG
Web site: http://www.octapharma.com/
