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Octapharma's Drive to Prevent and Eradicate FVIII Antibody Inhibitors in Haemophilia Treatment

A Panel of First- and Developing-World Experts Highlight Initiatives to Prevent and Treat FVIII Inhibitors Using the Human, Plasma-Derived, VWF-Containing FVIII Concentrate, Octanate®

LACHEN, Switzerland--(BUSINESS WIRE)--Sep 13, 2010 - In a showing of its continued commitment to eradicating today's major complication in the treatment of haemophilia with FVIII concentrates ˆ’ i.e. anti-FVIII neutralising antibodies ˆ’ Octapharma AG recently sponsored a symposium, "Inhibitors in Haemophilia A: Prevention, Current Management and Personalised Therapy Perspectives", on July 13, 2010 at the World Federation of Haemophilia's (WFH's) yearly congress. This year's location in Buenos Aires, Argentina, remarkably reflected Octapharma's steadfast interest in clinical benefit and cost effectiveness reaching the developing world. Octapharma's symposium brought together a consortium of international experts on blood coagulation disorders, each of them presenting on diverse topics relevant to the current and future management of patients with FVIII inhibitors, and on study results for the human, plasma-derived, von Willebrand factor (VWF)-containing FVIII concentrate, Octanate®.

“The development of FVIII antibody inhibitors during FVIII replacement is the most significant medical complication today in the life-long treatment of haemophilia," said Dr Olaf Walter, Head of Octapharma`s Haematology Business Unit. "As part of our mission to improve patient quality of life and treatment cost effectiveness, we supported this WFH symposium to highlight the major initiatives addressing treatment of patients with FVIII inhibitors.”

Neutralising FVIII antibody inhibitors arise most frequently in previously untreated severe haemophilia A patients (30% inhibitor rate). Octanate® has been successfully used by Octapharma for immune tolerance induction (ITI) therapy since 2006, and is the most proven treatment to eradicate FVIII inhibitors, re-establishing haemostasis via FVIII replacement in haemophiliacs. Dr Carmen Escuriola Ettingshausen (Johann-Wolfgang-Goethe University Hospital in Frankfurt, Germany), standing in for Dr Wolfhart Kreuz (also from Frankfurt), presented the community's newest data on Octanate® from the ongoing, investigator-initiated ObsITI (Observational Immune Tolerance Induction) study. ObsITI is currently the largest running study of ITI, with more than 100 documented patients, and is still recruiting. Further recruiting details are available at Dr Escuriola presented strong data for Octanate®, showing complete ITI success in 80% of the current 15 patients with the required 36-month follow-up. Complete success was observed after a mean of 12 months ITI therapy, and notably, all patients had at least one indicator for poor prognostic ITI outcome.

Dr Escuriola also presented data from the ongoing Octanate® GCP-PUP study and the study from the German Society of Thrombosis and Haemostasis (GTH) on the initial incidence of FVIII inhibitors in previously-untreated patients (PUPs), a group of patients considered high risk for developing inhibitors. Dr Escuriola indicated that in "patients with ‰¥ 50 exposure days, the inhibitor incidence was only 5.1% with Octanate®, compared with the overall rate of 17.3%,” and that “no inhibitors were observed in PUPs receiving prophylaxis with Octanate®.”

Dr Nadezhda Zozulya (Russian Academy of Medical Sciences in Moscow, Russia) presented data from a centre participating in the ObsITI study. “Eight out of ten adult patients achieved complete success with Octanate®,” said Dr Zozulya. “For all patients, the median number of bleeding episodes was reduced from 25 to 2.” Cost data from this adult cohort indicated a significant ‚¬5 million lifetime savings as compared to NovoSeven® by the age of 40 years, and there were statistically significant improvements in patients' quality of life.

The effects of FVIII inhibitors to bind particular regions (termed epitopes) of the FVIII protein in the ObsITI study was presented by Dr Erik Berntorp (Malmö University Hospital in Malmö, Sweden). Dr Berntorp discussed how Octanate® provokes less of an inhibitor reaction compared with recombinant FVIII products, by virtue of VWF protection of particular FVIII epitopes. He also indicated that "characterisation of inhibitors that recognise specific [FVIII] epitopes, known as epitope mapping, may facilitate individualisation of ITI treatment and tailoring strategies.” Dr Kate Khair from the Great Ormond Street Hospital for Children in London also suggested that Octanate® may be suitable for haemophiliacs with inhibitors who are “resistant” to FVIII replacement, having failed previous ITI with recombinant FVIII.

Of particular interest to the developing world, potential economic advantages of Octanate® were highlighted by Cesar Alberto Montaño (Hemophilia Treatment Center – Hemolife, Pereira, Colombia) who presented on Octanate® not only in ITI, but also in bleeding prophylaxis prior to inhibitor development. His remarkable data showed a $4.4 million and $8.1 million savings with Octanate® in ITI, as compared to NovoSeven® or FEIBA® on-demand by the age of 20 years. Dr Montaño maintained that in developing countries such as Colombia and Argentina, where significant achievements already are being made despite a short history of haemophilia care, the development of cost-effective therapies will be central to the effective management of patients with inhibitors.

About Octanate®

Octanate® is a human-derived, high-purity, VWF-stabilised, double virus-inactivated FVIII concentrate for intravenous administration. All coagulation FVIII present in Octanate® is bound to its natural stabiliser, VWF, in a VWF/FVIII ratio of approximately 0.4. Therefore, no additional stabilisers are required during the manufacture of Octanate®. Octanate® is safe, well-tolerated and efficacious in the treatment of bleeding in patients with all types of haemophilia A and is associated with a minimal risk of inhibitors in PUPs. Octanate® is highly successful in FVIII inhibitor eradication (ITI).

Octanate® is available in 250 IU, 500 IU and 1000 IU presentations. The mean specific activity of Octanate® is ‰¥ 100 IU/mg protein. Octanate® was first approved in Germany in August 1998 and has since been approved in 67 countries. Between August 1998 and February 2010, a total of more than 3.2 billion IU Octanate® have been distributed worldwide. .

About the XXIX 2010 Congress of the WFH in Buenos Aires, Argentina

The XXIX International Congress of the WFH is the largest event organised by the WFH in 2010, and is one of the most important events for the global haemophilia community. This year, more than 4,300 doctors, scientists, healthcare workers, patients with haemophilia, and representatives from haemophilia organisations gathered to learn about the latest developments in haemophilia treatment. Participants from 106 countries gathered at this record-breaking congress to present, discuss and debate the latest developments in haemophilia and related bleeding disorders.

About the Octapharma Group

Headquartered in Lachen, Switzerland, Octapharma is one of the largest plasma products manufacturers in the world and has been committed to patient care and medical innovation for over 27 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. Octapharma is currently developing the first commercial recombinant coagulation FVIII from a human cell line, which is designed to reduce the overall immunogenic challenge (and resulting inhibitor formation) to the haemophilia A patient.

At the present time, Octapharma has 37 subsidiaries and representative offices. The company employs over 4,000 people and is making sales in 80 countries worldwide. For more information, please visit

Contact: Octapharma
Corinne Landolt
Tel: +41 55 451 2136


Posted: September 2010