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- Publication of data from the WIL-31 study marks a milestone in improving long-term care for patients with severe von Willebrand disease (VWD)

LACHEN, Switzerland, January 22, 2024 /PRNewswire/ -- Octapharma today announced the publication of the results of the WIL-31 clinical study, which investigated the efficacy and safety of prophylaxis with wilate®, a von Willebrand factor/factor concentrate. VIII (VWF/FVIII), in patients with all types of von Willebrand disease (VWD). The results have been published in the leading medical journal Blood Advances [ Sidonio RF Jr et al. "Von Willebrand factor/factor VIII concentrate (wilate®) prophylaxis in children and adults with von Willebrand disease"]

The lead author and principal investigator of the WIL-31 study, Robert F. Sidonio Jr, M.D. (Medical Director of Hemophilia at the Aflac Cancer and Blood Disorders Center at Children's Healthcare of Atlanta and Associate Professor of Pediatrics at Emory University School of Medicine), commented: "WIL-31 is the largest prospective study to date. which examines the effectiveness and safety of regular prophylaxis in patients with VWD. Importantly, the study included a diverse patient population, including young children and adults, men and women, and all types of VWD. The WIL-findings 31 provide strong evidence supporting the use of VWF prophylaxis and have led to the recent addition of wilate® prophylaxis as a therapeutic indication for VWD in the US, expanding its access to the US patient population."

The WIL-31 study (NCT04052698) was a prospective, multinational, open-label, uncontrolled phase III study evaluating wilate® prophylaxis to prevent bleeding in VWD. Efficacy was evaluated in 33 adult and pediatric VWD patients aged 6 years or older from 14 centers in eight countries, and patients received wilate® prophylaxis two or three times a week for 12 months. All patients had participated in a 6-month prospective run-in study (WIL-29) during which they received on-demand treatment with any available VWF concentrate, allowing the efficacy of prophylaxis to be assessed at an intraindividual level.

The primary endpoint of the study, a >50% reduction in total mean annualized bleeding rate (ABR) during wilate® prophylaxis compared to pre-on-demand treatment, was met. The mean total ABR during wilate® prophylaxis was 5.2 compared to 33.4 during on-demand treatment, representing an 84% decrease. Mean spontaneous ABR decreased by 87% during wilate® prophylaxis compared to on-demand treatment. Reductions in total and spontaneous bleeding were comparable between age groups, bleeding sites and VWD disease types, and between men and women. Most patients received two doses per week and the mean weekly dose of wilate® was relatively low (58 IU/kg) compared to the dosage range recommended in the prescribing information.

Seven women of childbearing age were included in the study. This allowed the researchers to obtain information on the effect of wilate® prophylaxis on heavy menstrual bleeding, which is a major problem in women with VWD as there is limited clinical data on effective treatments. Although the data are based on a small sample size, they suggest that wilate® prophylaxis has a positive impact on reducing heavy menstrual bleeding; 71% (n=5) of patients do not experience heavy menstrual bleeding during wilate® prophylaxis.

Prophylaxis with wilate® was well tolerated in all types of VWD, in children and adults, and in men and women. During 12 months of wilate® prophylaxis, there were no serious wilate®-related adverse events or thrombotic events or FVIII accumulation.

"Unlike hemophilia A, prophylaxis is not a standard of care in VWD and less than 10% of patients with severe VWD receive prophylaxis," said Sigurd Knaub, senior vice president of CR

Octapharma would like to thank all participating centers, as well as patients and their caregivers, for their contribution to the study. Further publications on other analyzes of the WIL-31 study are planned.

About wilate®

wilate® is a high-purity human von Willebrand factor/factor VIII (VWF/FVIII) concentrate, which undergoes two virus inactivation steps during its production5. Albumin is not added as a stabilizer1. Purification processes result in a 1:1 ratio of VWF to FVIII that is similar to that of normal plasma1. wilate® contains a VWF triplet structure and a high molecular weight large multimer content similar to normal human plasma1. wilate® is available in presentations of 500 IU and 1000 IU.2

wilate® is indicated for the prevention and treatment of hemorrhage or surgical bleeding in von Willebrand disease (VWD), when desmopressin (DDAVP) alone is ineffective or contraindicated, and for the treatment and prophylaxis of hemorrhage in patients with hemophilia A (congenital factor VIII deficiency)2. The indication for prophylaxis in the USA is detailed in the updated prescribing information available here: wilate Full Prescribing Information (wilateusa.com)

About Octapharma

Headquartered in Lachen, Switzerland, Octapharma is one of the world's largest human protein manufacturers, 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 in three therapeutic areas: immunotherapy, hematology and critical care.

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

Octapharma press releases are directed specifically to the medical or healthcare media and not to the consumer press.

Contact:Ivana SpotakovaHead of Global CommunicationsIvana.Spotakova@octapharma.com 41 79 3474607

Referencias1.Stadler M et al. Biologicals 2006; 34:281–8.2.wilate® Summary of Product Characteristics 2023.

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