Octapharma USA Applies For Approval Of Wilate Supplements

Octapharma USA has submitted a Biologics License Application Supplement (sBLA) to the 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 Flemming Nielsen, President of Octapharma USA. "VWD patients urgently require treatment alternatives for von Willebrand disease prophylaxis. Octapharma is dedicated to providing life-saving therapies to the bleeding disorders community and medical professionals." 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 aged 6 and older with severe VWD of any type except type 2N, provides support for the application.

VWD is the most common bleeding disorder, affecting up to 1% of the population in the United States. Wilate is used to treat and control bleeding episodes in children and adults with VWD, as well as to manage bleeding during surgery. Furthermore, wilate is approved for routine prophylaxis in adolescents and adults with haemophilia A to reduce the frequency of bleeding episodes as well as 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.

“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.” During a previous six-month prospective, observational study, all WIL-31 patients received on-demand wilate treatment (WIL-29). WIL-31 was open to patients who had at least six bleeding episodes (BEs), excluding menstrual bleeds, with at least two of these BEs treated with a VWF-containing product. For 12 months, patients in WIL-31 received wilate prophylaxis two to three times per week at a dose of 20-40 IU/kg.

The primary goal of the clinical trials was to see if wilate prophylaxis reduced the mean total annualised bleeding rate (ABR) by more than 50% when compared to six months of on-demand treatment. Secondary objectives included measuring spontaneous ABR and treatment-emergent adverse events. During the previous study, researchers reported an 84% reduction in ABR when compared to on-demand treatment. The median spontaneous ABR fell 95%. During wilate prophylaxis, no serious drug-related adverse events or thrombotic events occurred.