Publication of Clinical Data Demonstrating Successful Immune Tolerance Induction (ITI) with OCTANATE® in Severe Haemophilia A in "Haemophilia"04.08.2015
Lachen, Switzerland August 4th 2015 - Octapharma announces the publication in Haemophilia of data reporting on successful Immune Tolerance Induction (ITI) with OCTANATE® in a large cohort of Haemophilia A patients with FVIII inhibitors. The data derive from an interim analysis of patients participating in the ongoing Observational Immune Tolerance Induction (ObsITI) study.
Development of inhibitors to FVIII products remains the most feared complication of Haemophilia A (HA) treatment, and publication of this highly favourable data on ITI with OCTANATE® marks a new milestone as the first-of-its-kind study for this well-established Octapharma product.
Data reporting on the progress and outcome in 48 patients who received Octapharma’s pdFVIII/VWF concentrate, OCTANATE®, as the sole FVIII product during treatment between December 2005 and October 2010 across 27 centres in 13 countries demonstrated a complete success rate of >70% with no relapses. A distinguishing quality of the ObsITI study is its inclusion of all eligible HA patients, regardless of historical ITI failure, or expected prognosis. The majority of patients – 40 of 48 (83.3%), exhibited one or more poor prognosis factors for ITI success (e.g. age ≥7 years, >2 years since inhibitor diagnosis, inhibitor titre ≥10 BU at the start of ITI, or prior ITI failure). Overall, inhibitor status became negative in 79.2% (38/48) patients. Notably, a significant 86% reduction in monthly bleeding rate was seen following successful inhibitor elimination, decreasing most dramatically during the initial 4 months of ITI.
These data from the ObsITI study with OCTANATE® underline the clinical benefit and long-term economic value of undertaking ITI in HA patients with inhibitors, regardless of the presence of factors historically considered to represent a ‘poor prognosis’ for success. Successful immune tolerance allows resumption of a standard prophylactic treatment regimen, guarding against long-term joint damage, arthropathy and morbidity for our patients. It has long been postulated that the presence of physiological levels of von Willebrand Factor in plasma-derived FVIII concentrates plays a beneficial role in ITI, and these data with OCTANATE® further support this claim.
Larisa Belyanskaya, Head of Octapharma’s International Business Unit for Haematology, said "Publication of interim data with OCTANATE® from this extensive independent, international, observational study has been eagerly awaited and we are now delighted to be able to share the outstanding success of OCTANATE® in the ObsITI study with a wider audience."
About ObsITI study
The ObsITI study, initiated in 2005, is an ongoing, international observational study co-ordinated by Dr. Carmen Escuriola-Ettingshausen (HZRM, Frankfurt-Mörfelden, Germany). ObsITI allows a systematic evaluation of ITI in haemophilia A patients with inhibitors and potential predictors of ITI outcome and morbidity.
OCTANATE® is a human plasma‑derived, high‑purity, freeze‑dried, double virus‑inactivated FVIII concentrate for intravenous administration. Coagulation FVIII present in OCTANATE® is bound to its natural stabiliser, von Willebrand factor (VWF), in a VWF/FVIII ratio of approximately 0.4. Therefore, no additional stabilisers are required during manufacturing.
OCTANATE® is used in the prophylaxis and treatment of bleeding in patients with all types of haemophilia A. OCTANATE® is available in 250 IU, 500 IU and 1000 IU presentations. OCTANATE® was first launched in Germany in 1998 and has since been approved in 83 countries. Since its launch, a total of approximately 8 billion IU OCTANATE® have been distributed worldwide. OCTANATE® is indicated for ITI in 32 countries.
About Haemophilia A
Haemophilia A is an X-linked hereditary disorder caused by FVIII deficiency which if left untreated leads to haemorrhages in muscles and joints and consequently to arthropathy and severe morbidity. FVIII replacement prophylactic treatment reduces the number of bleeding episodes and the risk of permanent joint damage. This disorder affects one in every 5,000 to 10,000 men worldwide. Globally, 75% of haemophilia cases are left undiagnosed or untreated. The development of neutralising FVIII antibodies (FVIII inhibitors) against infused FVIII represents the most serious treatment complication. The cumulative risk of FVIII inhibitor development is reported to be currently up to 38%.
About Octapharma in Haemophilia
Octapharma has been dedicated to improving haemophilia care for over 30 years. Addressing the challenge of inhibitor formation in haemophilia A has been the focus of our haemophilia product development as well as our research efforts. For over a decade Octapharma has invested in inhibitor management registry studies, and in recombinant development. Our understanding of bleeding disorders is reflected not only in our products for the treatment of haemophilia A, haemophilia B and von Willebrand disease, but also in the development of personalised treatment options.
Headquartered in Lachen, Switzerland, Octapharma is one of the largest human protein product manufacturers in the world and has been committed to patient care and medical innovation since 1983. Its core business is the development and production of human proteins from human plasma and human cell-lines. Octapharma employs approximately 6,000 people worldwide to support the treatment of patients in over 100 countries with products across the following therapeutic areas:
• Haematology (coagulation disorders)
• Immunotherapy (immune disorders)
• Critical Care
Octapharma owns five state-of-the-art production facilities in Austria, France, Germany, Sweden and Mexico.