FDA Confirms Orphan Drug Exclusivity Approval for wilate®, Octapharma USA's New Therapy for Common Inherited Bleeding Disorder

HOBOKEN, N.J., Aug. 2 /PRNewswire/ -- Octapharma USA has received confirmation of orphan drug exclusivity from the U.S. Food and Drug Administration (FDA) for wilate® (von Willebrand Factor/Factor VIII Concentrate, Human), the replacement therapy developed specifically for von Willebrand Disease (VWD). The approval comes from the FDA Office of Orphan Products Development, which helps to advance the development of products that demonstrate promise for the treatment of rare diseases.

"In awarding orphan drug exclusivity, the FDA Office determined that wilate® offers greater viral safety than Humate-P," said Flemming Nielsen, President of Octapharma USA, the rapidly growing U.S. division of Octapharma AG, one of the world's largest human protein products manufacturers. "wilate® has received seven years of marketing exclusivity as a result of the evidence suggesting wilate® is as efficacious and safer than Humate P."

The FDA has approved wilate® for the treatment of spontaneous or trauma-induced bleeding episodes in patients with severe VWD as well as in patients with mild or moderate forms of the illness 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) step. The selected purification processes isolates the VWF/FVIII complex under highly protein-protecting conditions, resulting in a 1:1 ratio of VWF:RCo (ristocetin cofactor) to FVIII activity 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.  wilate® contains a VWF triplet structure and VWF mulitmeric distribution similar to normal human plasma.

According to the National Institutes of Health, VWD is the most common inherited bleeding disorder and occurs in about 1 out of every 100 to 1,000 people. "The FDA orphan drug exclusivity approval for wilate® confirms Octapharma's decision to focus exclusively on the treatment of von Willebrand patients and further demonstrates Octapharma's commitment to the continued advancement of human protein therapies," said Nielsen.

For more information on wilate®, please visit www.wilateusa.com.

About the Octapharma Group

Headquartered in Lachen, Switzerland, Octapharma is one of the largest human protein products manufacturers in the world and has been committed to patient care and medical innovation for over 27 years. 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's wilate® received orphan drug exclusivity from the U.S. Food and Drug Administration (FDA) for the treatment of von Willebrand disease (VWD). Octapharma employs over 4,000 people and has biopharmaceutical experience in 80 countries worldwide, including the United States, where Octapharma USA is located in Hoboken, N.J.  Octapharma operates two state-of-the-art production sites licensed by the FDA, providing a high level of production flexibility. For more information, please visit www.octapharma.com.

Forward-looking statements

This news release contains forward-looking statements, which include known and unknown risks, uncertainties and other factors not under the company's control. The company assumes no liability whatsoever to update these forward-looking statements or to conform them to future events or developments. These factors include results of current or pending research and development activities and actions by the FDA or other regulatory authorities.

SOURCE Octapharma USA