Octapharma sponsors symposium on inhibitors in Haemophilia A

Sep 13 2010

In a showing of its continued commitment to eradicating today's major complication in the treatment of haemophilia with FVIII concentrates − i.e. anti-FVIII neutralising antibodies − 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^®.

“Eight out of ten adult patients achieved complete success with 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 www.obsiti.com. 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.

SOURCE Octapharma Group