Octapharma to present clinical and scientific data at the 17th Annual Congress of the European Association for Haemophilia and Allied Disorders

Octapharma to present clinical and scientific data that advances our understanding and treatment of bleeding disorders at the 17^th Annual Congress of the European Association for Haemophilia and Allied Disorders (EAHAD), which takes place in Frankfurt, Germany, from February 6 – 9, 2024.

• Octapharma will present new clinical and scientific findings for wilate^® and Nuwiq^®

• New findings in von Willebrand disease (VWD) and haemophilia A, will be presented by leading experts in a Satellite Symposium, as well as an oral and six poster presentations

• Presentations will include new data on the efficacy and safety of wilate^® prophylaxis in people with von Willebrand disease (VWD)

Showcasing wilate^® at EAHAD 2024

WIL-31 is the largest prospective prophylaxis study in VWD with an on-demand run-in study as an intra-individual patient comparator. The study shows that wilate^® prophylaxis is highly effective at reducing bleeding rates in children and adults with all types of VWD and across examined bleeding sites. The results provide compelling evidence for the use of regular prophylaxis in people with VWD.

“The new data from the WIL-31 study provide strong evidence for the use of wilate^® prophylaxis in people with all types of VWD. The findings have led to the approval of wilate^® as prophylaxis in the US, thereby expanding the therapeutic options for these patients.”

Larisa Belyanskaya, Senior Vice President and Head of IBU Haematology

A deep-dive into the findings from the WIL-31 study will be showcased during Octapharma’s Satellite Symposium ‘Shifting prophylaxis paradigms in VWD: The WIL-31 study in focus’:

• Jan Astermark (Sweden) will explore the underutilisation of prophylaxis in VWD compared to haemophilia A

• Robert F. Sidonio Jr. (US) will discuss how the findings from WIL-31 have challenged the status quo of prophylaxis in VWD

• The efficacy of wilate^® prophylaxis will be explored in three interactive cases of patients in the WIL-31 study:

  • Child living with VWD presented by Robert F. Sidonio Jr. from Atlanta

  • Adult living with frequent nosebleeds presented by Ana Boban from Zagreb

  • Female experiencing heavy menstrual bleeding presented by Csongor Kiss from Debrecen

The Satellite Symposium will take place on Wednesday, February 7, 17:30–18:45 (CET), in Panorama Room 2.

The importance of preventing frequent nosebleeds in people with VWD, and the efficacy of wilate^® prophylaxis in this setting, will be presented by Ana Boban in the oral SLAM presentation ‘Regular prophylaxis with a plasma-derived von Willebrand factor/factor VIII concentrate is effective for reducing nosebleeds in children and adults with von Willebrand disease’ (OR04) on Friday, February 9, 08:30–10:00 CET, Session 6 SLAM.

Two poster presentations provide additional insights into the findings of the WIL-31 study:

• PO213: No accumulation of factor VIII and von Willebrand factor during prophylaxis with a plasma-derived von Willebrand factor/factor VIII concentrate during the WIL-31 study

• PO194: Managing von Willebrand disease with inhibitors during prophylaxis with a plasma derived von Willebrand factor/factor VIII concentrate – The WIL-31 study

Showcasing Nuwiq^® at EAHAD 2024

Beyond its role in the coagulation cascade, emerging evidence indicates that FVIII influences platelet and endothelial cell function. Replacement therapy with recombinant FVIII (rFVIII) concentrates has become a mainstay of treatment for people with haemophilia A; however, it is unknown if there is a difference in how modifications of rFVIII concentrates impact platelet and endothelial cell functionality. Binding of Nuwiq^® to activated platelets in vitro was found to be greater compared with other rFVIII concentrates, while in separate research Nuwiq^® showed stronger effects on endothelial cell function compared with other rFVIII concentrates:

• PO027: Impact of differential binding of recombinant factor VIII concentrates to platelets on platelet functionality

• PO024: Investigating the role of factor VIII in endothelial cell function

In rare diseases such as haemophilia A, it is not possible to directly compare treatments in a clinical study. In such instances, indirect treatment comparisons can be used to compare the effects of different treatments. One such well-established indirect comparison method is matching-adjusted indirect comparison (MAIC). MAICs were used to compare the efficacy of personalised, pharmacokinetic-guided prophylaxis with Nuwiq^® versus other treatments:

• PO041: Personalised prophylaxis with simoctocog alfa versus standard prophylaxis with efanesoctocog alfa in haemophilia A, a matching-adjusted indirect comparison

• PO042: Personalised prophylaxis with simoctocog alfa versus standard emicizumab prophylaxis in haemophilia A, a matching-adjusted indirect comparison

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.

Octapharma employs more than 11,000 people worldwide to support the treatment of patients in 118 countries with products across three therapeutic areas: Immunotherapy, Haematology and Critical Care.

Octapharma has seven R&D sites and five state-of-the-art manufacturing facilities in Austria, France, Germany and Sweden, and operates more than 190 plasma donation centres across Europe and the US. Octapharma has 40 years of experience in patient care.

About Nuwiq^® 

Nuwiq^® (simoctocog alfa) is a 4^th generation recombinant factor VIII (rFVIII) protein, produced in a human cell line without chemical modification or fusion with any other protein¹. It is cultured without additives of human or animal origin, is devoid of antigenic non-human protein epitopes and has a high affinity for von Willebrand factor¹.

Nuwiq^® treatment has been assessed in nine^1-3 completed clinical trials which included 201 previously treated patients (190 individuals)¹ and 108 previously untreated patients² with severe haemophilia A.

Nuwiq^® is available in 250 IU, 500 IU, 1,000 IU, 1,500 IU, 2,000 IU, 2,500 IU, 3,000 IU and 4,000 IU presentations⁴. Nuwiq^® is approved for use in the treatment and prophylaxis of bleeding in patients with haemophilia A (congenital FVIII deficiency) across all age groups⁴. 

About wilate^® 

wilate^® is a high-purity human von Willebrand factor/factor VIII (VWF/FVIII) concentrate, that undergoes two virus inactivation steps during its production⁵. No albumin is added as a stabiliser⁵. The purification processes result in a 1:1 ratio of VWF to FVIII that is similar to normal plasma⁵. wilate^® contains a VWF triplet structure and content of large high molecular weight multimers similar to normal human plasma⁵.

wilate^® is available in 500 IU and 1,000 IU presentations.⁶ wilate^® is indicated for the prevention and treatment of haemorrhage or surgical bleeding in von Willebrand disease (VWD), when desmopressin (DDAVP) alone is ineffective or contra-indicated, and for the treatment and prophylaxis of bleeding in patients with haemophilia A (congenital factor VIII deficiency)⁶. 

The indication for prophylaxis in the US is detailed in the updated prescribing information available here.

Octapharma press releases are specifically for health specialist/medical media and are not for consumer press.  

References:

1. Lissitchkov T et al. Ther Adv Hematol 2019; 10:2040620719858471. 

2. Liesner RJ et al. Thromb Haemost 2021; 121:1400-8. 

3. Octapharma AG; Data on file. 

4. Nuwiq^® Summary of Product Characteristics. 

5. Stadler M et al. Biologicals 2006; 34:281-8. 

6. wilate^® Summary of Product Characteristics.