Octapharma Symposium At ISTH Berlin Supports Progress in Lifelong Treatment of High-Risk Patients With Von Willebrand Disease (VWD) and haemophilia A

Octapharma is pleased to announce the success of yesterday’s symposium at the International Society on Thrombosis and Haemostasis (ISTH) congress held in Berlin, Germany. As platinum sponsors of the congress, Octapharma is reflecting its dedication to bleeding disorder research with two diverse scientific symposia in von Willebrand disease (VWD) and haemophilia A (HA). Following on from a successful symposium on Nuwiq^® (the company’s most recent addition to their expanding haematology portfolio), the latest developments with wilate^®, a high purity, double virus-inactivated VWF/FVIII concentrate containing both factors in a physiological 1:1 ratio, were discussed on Monday 10^th July in a symposium entitled, “wilate ^® for a Lifetime of Care, Bleeding Management across High-Risk Patient Populations”. The symposium, chaired by Professor Ute Scholz (Centre of Coagulation disorders, Leipzig, Germany) addressed the life-long treatment of high-risk patients, including those with FVIII or VWF inhibitors, women with VWD during pregnancy and childbirth, and ageing VWD patients.

The session began with Stacy Croteau (Boston, USA) discussing her unique experience with the treatment of two boys with neutralising alloantibodies against VWF in a talk entitled “Immune tolerance induction for neutralising VWF alloantibodies”. Both boys underwent immune tolerance induction (ITI) treatment with wilate ^® and additional immunosuppression in an adapted ITI protocol. Sharing this experience is extremely valuable for the VWD community because, although very rare in VWD, the occurrence of inhibitors is one of the greatest challenges to the management of bleeding disorders, with no established treatment for eradication of inhibitors in VWD patients.

Jill Johnsen (Seattle, USA) followed with a presentation entitled “Challenges in pregnancy and delivery in VWD – Need for a new post-partum bleeding study”, which highlighted the demand to increase our understanding of VWD in women during pregnancy and childbirth. VWF is markedly elevated during normal pregnancy and current guidelines for management of the disease during labour and delivery remain very variable internationally and are not always sufficient. Dr Johnsen gave an overview of published data in this field and proposed a new study investigating the risks of post-partum bleeding in women with VWD, highlighting the need to find ways to improve outcomes for such patients.

John Pasi (London, UK) discussed the rising number of elderly patients with VWD, and the associated challenges in his presentation, “Ageing VWD patients – shifting focus to an emerging population”. Elderly VWD patients often have the additional complications of co-morbidities and other medication. Professor Pasi discussed the potential benefits using wilate^® from his own experience with treating such patients: the physiological FVIII/VWF ratio simplifies dosing and monitoring and can reduce the physician’s anxiety regarding thrombosis risk, which is a greater concern in the elderly.

Finally, Robert Klaassen (Ottawa, Canada) presented the Canadian experience with wilate^® in patients with HA. His presentation, “wilate ^® in haemophilia A – clinical experience in PUPs and ITI” included data demonstrating the efficacy and safety of wilate^® in ITI for patients with HA and inhibitors: inhibitor levels in 10 of 12 of the patients receiving ITI with wilate^® were reduced to undetectable levels. A new Canadian Study, PREVAIL, will further investigate the efficacy of wilate^® in patients with HA and inhibitors. The Canadian experience with wilate^® offers encouraging evidence for its use in ITI. Dr Klaassen further supported these potential benefits of wilate^® with promising data from ITI in one of his patients.

The focus on patient cases from real-life practice in this symposium highlighted the issues for consideration when treating specific subgroups of VWD and haemophilia A patients; the data from studies using wilate^® strengthened the evidence for the significant contribution of this naturally balanced VWF/FVIII complex concentrate in bleeding management.

Olaf Walter, Board Member at Octapharma, described the session as “impactful and well-received”, commenting that “Octapharma is very pleased with the outcome of the wilate ^® ISTH symposium, once again we were able to demonstrate our dedication and commitment to patients in addressing unmet needs and advancing the effective treatment of bleeding disorders”.

About von Willebrand Disease

VWD is the most common of the inherited bleeding disorders, with approximately 1% of the population having VWF levels below normal. VWD is classified as type 1 (generally mild), type 2 (variable) or type 3 (severe). The symptoms of VWD are usually those of platelet dysfunction and include nose bleeds, skin bruises and haematomas, prolonged bleeds from trivial wounds, oral cavity bleeding, and excessive menstrual bleeding. Gastrointestinal bleeds are relatively rare, but may be very serious when they occur. Severe deficiency of VWF, or a specific defect in the interaction of VWF with FVIII, causes a secondary moderate deficiency of FVIII. These patients may have symptoms that are more characteristic of haemophilia, such as bleeds into joints or soft tissues including muscle and brain.

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 neutralizing 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 39%.

About wilate ^®

wilate^® is a high-purity, plasma-derived human von Willebrand factor/coagulation factor VIII complex concentrate in a naturally balanced 1:1 activity ratio. wilate^® is exclusively derived from human plasma collected in U.S. FDA-approved plasma donation centres. The production of wilate^® includes two independent state-of-the-art virus inactivation steps: Solvent/Detergent (S/D) treatment irreversibly inactivates lipid-enveloped viruses by destroying their lipid coat, while PermaHeat treatment inactivates both enveloped and non-enveloped viruses. The addition of albumin for stabilisation is not necessary with wilate^®. The excellent clinical efficacy, safety, and tolerability of wilate^® both in the treatment and prevention of bleeding, as well as in surgical prophylaxis, have been proven in prospective controlled trials and post-marketing studies. wilate^® is approved in over 60 countries worldwide and in most countries is indicated for the treatment and prophylaxis of children and adults with von Willebrand disease and haemophilia A.

About Octapharma

Headquartered in Lachen, Switzerland, Octapharma is one of the largest human protein manufacturers in the world, developing and producing human proteins from human plasma and human cell lines. As a family-owned company, Octapharma believes in investing to make a difference in people’s lives and has been doing so since 1983; because it’s in our blood.

In 2016, the Group achieved €1.6 billion in revenue, an operating income of €383 million and invested €249 million to ensure future prosperity. Octapharma employs more than 7,100 people worldwide to support the treatment of patients in 113 countries with products across three therapeutic areas:

· Haematology (coagulation disorders)

· Immunotherapy (immune disorders)

· Critical care

Octapharma owns six state-of-the-art production facilities in Austria, France, Germany, Mexico and Sweden.

For more information visit www.octapharma.com

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Contact information

Octapharma AG
International Business Unit- Haematology
Olaf Walter
Olaf.Walter@octapharma.com
or
Larisa Belyanskaya
Larisa.Belyanskaya@octapharma.com
Tel: +41 55 4512121