LACHEN, Switzerland--(BUSINESS WIRE)--Octapharma will sponsor a range of activities at this year’s WFH World Congress in Orlando, USA, including two symposia and a number of posters and scientific presentations highlighting Octapharma’s recent developments in haemophilia A and von Willebrand disease (VWD).
The first symposium, “Simple solutions to complex issues: Addressing today’s challenges in VWD and haemophilia A” on Monday 25 July 2016, will be chaired by Professor Erik Berntorp (Lund University, Malmö, Sweden) and will explore current clinical issues in the management of patients with VWD and haemophilia A. New insights into prophylactic therapy for VWD patients will be discussed, including the management of bleeding in patients in the perioperative setting, and special issues related to VWD in women will be addressed. The latest clinical experiences with wilate®, a high purity, double virus-inactivated VWF/FVIII concentrate containing both factors in a physiological 1:1 ratio, will be presented including its use in immune tolerance induction in haemophilia A patients with FVIII inhibitors.
Major challenges in current haemophilia A treatment include the development of inhibitors and the need for frequent venous access for FVIII injection. Octapharma’s human cell line recombinant FVIII Nuwiq® (Human-cl rhFVIII)®, is a fourth generation rFVIII derived from a human cell line without chemical modification or fusion with any other protein and is devoid of antigenic non-human epitopes1,2. The development aim of Nuwiq® is to address the challenges of FVIII inhibitors and frequent infusions required during prophylaxis.
The second symposium, “A focus on human cell-line derived rFVIII in haemophilia A: Update on clinical experience with Nuwiq® in PUPs and PTPs” on Wednesday 27 July, will be chaired by Professor Craig Kessler (Georgetown University Medical Centre, Washington DC, USA) and will provide an opportunity to discuss the current complex issues in haemophilia A and the latest experience with Nuwiq®. The risk of inhibitor development throughout the life of haemophilia A patients, taking into account factors such as age and the need for intensive treatment during surgery will be reviewed. New interim data from a clinical study of Nuwiq® in previously untreated patients (PUPs) will be presented, as well as the importance of PK-guided personalised prophylaxis and its potential to reduce the number of infusions and bleeding rates.
In addition, the following scientific posters will be presented during the congress:
- Individualized prophylaxis with Nuwiq® (Human-cl rhFVIII) in previously treated adults with severe haemophilia A who had received regular routine prophylaxis in the past
- PUPs with Severe Haemophilia A - A GCP Study Evaluating the Immunogenicity, Efficacy and Safety of Nuwiq® (NuProtect)
- Individual baseline thrombin generation and bleeding rate during personalized prophylaxis with Nuwiq® in previously treated patients with severe haemophilia A
- Long-term Immunogenicity, Safety and Efficacy of Human-cl rhFVIII in Previously Treated Children with Severe Haemophilia A
- Experience with Nuwiq® in clinical trials with previously treated paediatric and adult patients
- Surveillance Study of Safety and Efficacy of a VWF/FVIII concentrate in patients with von Willebrand disease (VWD) – data report from an ongoing study (Wil-20)
- Immune tolerance induction in haemophilia A inhibitor patients with a single factor VIII/von Willebrand factor concentrate in an observational immune tolerance induction study (ObsITI)
- Disease severity as a predictor of response to immune tolerance induction (ITI) in adult patients with haemophilia A and high titer inhibitors
- Pharmacokinetic (PK) Comparison of Two Fibrinogen Concentrates in Patients with Congenital Fibrinogen Deficiency: final analysis.
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 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%.
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.
Octapharma employs approximately 6,400 people worldwide to support the treatment of patients in 105 countries with products across three therapeutic areas:
• Critical Care
• Haematology (coagulation disorders)
• Immunotherapy (immune disorders)
Octapharma owns five state-of-the-art production facilities in Austria, France, Germany, Mexico and Sweden.
For more information visit www.octapharma.com
1. FDA Memorandum to Octapharma dated 09 October 2014, reference STN 125555\0
2. Kannicht et al. Thrombosis Research 2013; 131: 78–88