Swiss-based Octapharma AG sponsored two symposia during the International Society of Thrombosis and Haemostasis (ISTH) Biennial Congress in Boston, July 11-16, 2009. Gathering for the events were many prominent international blood coagulation disorder researchers.
The first symposium, “Prevention and Eradication of FVIII Inhibitors: Bridging Lab and Field Research” was chaired by David Lillicrap, MD, Professor of Pathology and Molecular Medicine at Queen’s University in Ontario, Canada, and Georges E. Rivard, MD, Professor of Pediatrics at Université de Montréal. The researchers discussed recent data supporting the use of von Willebrand factor (VWF)/FVIII concentrates for immune tolerance induction in hemophilia A patients with poor prognosis for a successful ITI outcome.
“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,” said Octapharma AG Vice Chairman Kim Björnstrup. “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 second symposium, “From Humans to Humans--Introducing the First Recombinant FVIII Produced from a Human Cell Line,” was chaired by Edward G.D. Tuddenham, MD, Director of The Katharine Dormandy Haemophilia Center & Thrombosis Unit in London, and Johannes Oldenburg, MD, PhD, Chairman and Director of the Institute of Experimental Haematology and Transfusion Medicine in Bonn, Germany. 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.
“Twenty years after the start of clinical trials with rFVIII concentrates expressed by hamster cells, a new rFVIII compound has recently entered clinical studies,” said Tuddenham. “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.”
Source: Octapharma press release dated August 31, 2009