Octapharma USA Requests FDA Approval for wilate® VWD Prophylaxis Supplement

• Biologics License Application Supplement supported by study evaluating efficacy and safety of wilate^® prophylaxis in adults and children age six and older with all types of VWD
• Researchers reported an 84% reduction in annual bleeding rate and 95% decrease in median spontaneous annual bleeding rate when compared with on-demand treatment during a prior study
• No serious drug-related adverse events or thrombotic events occurred

PARAMUS, N.J., Feb. 23, 2023 /PRNewswire/ -- Octapharma USA has submitted a Biologics License Application Supplement (sBLA) to the U.S. Food and Drug Administration (FDA) to expand the approval of wilate^®, von Willebrand Factor/Coagulation Factor VIII Complex (Human) Lyophilized Powder for Solution for Intravenous Injection, to include routine prophylaxis to reduce the frequency of bleeding episodes in children and adults with any type of von Willebrand disease (VWD).

"We look forward to working with the FDA on this sBLA for wilate^® prophylaxis in children and adults," said Octapharma USA President Flemming Nielsen. "People with VWD urgently need treatment alternatives for von Willebrand disease prophylaxis. Octapharma is committed to delivering life-saving therapies to the bleeding disorders community and medical providers."

The application is supported by the results of Octapharma's WIL-31 study, a prospective, non-controlled, international, multicenter phase 3 trial that investigated the efficacy and safety of wilate^® prophylaxis over 12 months in people of age 6 and older with severe VWD of any type except type 2N. VWD is the most common bleeding disorder, found in up to 1% of the U.S. population.¹

Wilate^® is indicated in children and adults with VWD for on-demand treatment and control of bleeding episodes and perioperative management of bleeding. Additionally, wilate^® is indicated in adolescents and adults with hemophilia A for routine prophylaxis to reduce the frequency of bleeding episodes and on-demand treatment and control of bleeding episodes.

"Long-term prophylaxis with wilate^® met study endpoints in pediatric and adult patients with VWD of all types except type 2N during our clinical trial," said WIL-31 Principal Investigator Robert F. Sidonio Jr., M.D., Medical Director of Hemophilia, Aflac Cancer & Blood Disorders Center with Children's Healthcare of Atlanta and Associate Professor of Pediatrics, Emory University School of Medicine.² "I am surprised at the number of bleeding episodes patients have been living with while receiving on-demand treatment and impressed how much their quality of life can be improved with prophylaxis."

All WIL-31 patients received on-demand treatment with wilate^® during a previous six-month, prospective, observational study (WIL-29). Patients who experienced at least six bleeding episodes (BEs), excluding menstrual bleeds, with at least two of these BEs treated with a VWF-containing product, were eligible to enter WIL-31. Patients in WIL-31 received wilate^® prophylaxis, two to three times per week at a dose of 20-40 IU/kg, for 12 months. The clinical trial's primary purpose was to investigate whether prophylaxis with wilate^® lowered the mean total annualized bleeding rate (ABR) compared to the six months of on-demand treatment by more than 50%. Secondary goals were to measure spontaneous ABR and treatment-emergent adverse events.²

Researchers reported an 84% reduction in ABR compared with on-demand treatment during the prior study. The median spontaneous ABR decreased by 95%. No serious drug-related adverse events or thrombotic events occurred during prophylaxis with wilate^®.²

About wilate^®
Wilate^®, von Willebrand Factor/Coagulation Factor VIII Complex (Human) Lyophilized Powder for Solution for Intravenous Injection is indicated in children and adults with von Willebrand disease for on-demand treatment and control of bleeding episodes and perioperative management of bleeding. Wilate^® is indicated in adolescents and adults with hemophilia A for routine prophylaxis to reduce the frequency of bleeding episodes and on-demand treatment and control of bleeding episodes.

CONTRAINDICATIONS
Do not use in patients with known hypersensitivity reactions, including anaphylactic or severe systemic reaction, to human plasma-derived products, any ingredient in the formulation, or components of the container.

WARNINGS AND PRECAUTIONS
Anaphylaxis and severe hypersensitivity reactions are possible. Thromboembolic events may occur. Monitor plasma levels of FVIII activity. Development of neutralizing antibodies to FVIII and to VWF, especially in VWD type 3 patients, may occur. Wilate^® is made from human plasma and carries the risk of transmitting infectious agents.

For complete prescribing information, please visit wilateusa.com.

About the Octapharma Group
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 around 10,000 people worldwide to support the treatment of patients in 118 countries with products across three therapeutic areas: Immunotherapy, Hematology, and Critical Care. Octapharma has seven research and development sites and five state-of-the-art manufacturing facilities in Austria, France, Germany and Sweden, and operates more than 180 plasma donation centers across Europe and the U.S. The company's American subsidiary, Octapharma USA, is located in Paramus, N.J. For more information, please visit octapharmausa.com.

REFERENCES
1 – Centers for Disease Control and Prevention website, Data and Statistics on von Willebrand Disease, accessed Feb. 3, 2023.
2 – Robert F. Sidonio, Jr., Ana Boban, Leonid Dubey, Adlette Inati, Csongor Kiss, Toshko Lissitchkov, Dzmitry Novik, Elina Peteva, Ali T. Taher, Margarita Arkadevna Timofeeva, Kateryna V. Vilchevska, Vladimir Vdovin, Sylvia Werner, Sigurd Knaub, Claudia Khayat; Efficacy and Safety of Prophylaxis with a Plasma-Derived Von Willebrand Factor/ Factor VIII Concentrate in Previously Treated Patients with Von Willebrand Disease. Blood 2022; 140 (Supplement 1): 8438–8439. doi: https://doi.org/10.1182/blood-2022-162358.

WIL-0484-PPR

Octapharma USA, Inc. • 117 West Century Road • Paramus • NJ 07652 • 201-604-1130 • www.octapharmausa.com 

SOURCE Octapharma USA