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.
PARAMUS, N.J., Feb. 23, 2023 /PRNewswire/ -- Octapharma USA, Inc. 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).
Octapharma USA seeks FDA approval for wilate® for routine prophylaxis to reduce the frequency of VWD bleeding episodes “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® CONTRAINDICATIONS WARNINGS AND PRECAUTIONS For complete prescribing information, please visit wilateusa.com. About the Octapharma Group REFERENCES WIL-0484-PPR Octapharma USA, Inc. • 117 West Century Road • Paramus • NJ 07652 • 201-604-1130 • www.octapharmausa.com
SOURCE Octapharma USA |
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