wilate to treat bleeding disorder von Willebrand disease to be launched in US: Octapharma

wilate, the first replacement therapy for von Willebrand disease, has been given orphan drug status approval in US, announced Octapharma.

wilate has been approved for the treatment of spontaneous and trauma-induced bleeding episodes in patients with all types of von Willebrand disease (VWD), by the US FDA.

wilate is a double virus inactivated von Willebrand Factor/Coagulation Factor VIII Concentrate (Human).

wilate has demonstrated efficacy for all types of VWD including pediatric patients in four prospective clinical trials utilizing both objective and subjective data, according to Octapharma.

wilate is indicated for the treatment of spontaneous and trauma-induced bleeding episodes in patients with severe VWD as well as patients with mild or moderate VWD in whom the use of desmopressin is known or suspected to be ineffective or contraindicated.

wilate is the first double virus inactivated VWF/FVIII (von Willebrand Factor / Factor FVIII), high-purity concentrate, utilizing the solvent/detergent (S/D) process and a special terminal dry-heating (TDH) system.

The selected purification processes isolates the VWF/FVIII complex under highly protein-protecting conditions, resulting in an approximately 1:1 ratio of VWF:RCo (ristocetin cofactor) and FVIII activities that is similar to normal plasma. No albumin is added as a stabilizer.

wilate is exclusively derived from large pools of human plasma collected in U.S. FDA approved plasma donation centers.

Octapharma plans to launch wilate in US by early 2010.

von Willebrand disease is the most common bleeding disorder, which is found in approximately 1% to 2% of the U.S. population, according to the Centers for Disease Control and Prevention.

von Willebrand disease is a result of the body’s inability to make von Willebrand Factor, the human protein that helps clot blood.

“The FDA approval of wilate makes this the first and only replacement therapy developed and manufactured specifically for VWD. Its unique viral attenuation steps and it’s close to 1:1 ratio of FVIII and VWF will provide a next generation treatment option for patients with von Willebrand disease,” stated Craig Kessler, M.D., Georgetown University Hospital, Professor of Medicine and Pathology and Director of the Division of Coagulation.

Four prospective clinical trials have demonstrated the safety, tolerability and hemostatic efficacy of wilate in the treatment of acute bleeding episodes and prophylaxis in patients with various types of VWD.

Wilate was observed in 1,068 bleeding episodes and determined to be successful between 84% and 93% of the time with results varying dependent on patient type, the company said.

Since the mid-1980s, the requirements for the viral safety of plasma preparations have constantly been made increasingly stringent, requiring demonstrated virus elimination/inactivation.

Several viral inactivation steps have enhanced the safety of coagulation products, but S/D inactivation is the current gold standard for safety from highly infectious enveloped viruses.

Octapharma was the first manufacturer to apply the S/D inactivation to a large-scale production of plasma derivatives.

The wilate manufacturing process provides two independent and effective virus inactivation procedures, namely S/D treatment in bulk and TDH treatment of the lyophilized product in final container. In addition, the ion-exchange chromatography step utilized during wilate manufacturing contributes to the viral safety.

Plasma contains VWF and FVIII at very low concentration. The Wilate manufacturing process is designed to enrich the proportion of VWF/FVIII complex. Accompanying plasma proteins that may give rise to clinical side-effects, as well as proteases that could impair the stability of coagulation factors and degrade their natural structure and functionality, are efficiently removed during production.

Headquartered in Lachen, Switzerland, Octapharma is one of the largest plasma products manufacturers in the world.

Octapharma’s core business is the development, production and sale of high quality human protein therapies from both human plasma and human cell lines, including immune globulin intravenous (IGIV).

In the U.S., Octapharma’s IGIV product, octagam (immune globulin intravenous [human] 5%), is used to treat disorders of the immune system, and Octapharma’s Albumin (Human) is indicated for the restoration and maintenance of circulating blood volume.

Octapharma operates two state-of-the-art production sites licensed by the U.S. Food and Drug Administration.

Octapharma USA is the U.S. division of Octapharma AG.