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FDA Approves Wilate

Octapharma USA Announces FDA Approval of Wilate - the First Replacement Therapy Developed Specifically for von Willebrand Disease

HOBOKEN, N.J., Dec. 7 /PRNewswire/ -- Octapharma USA today announced the U.S. Food and Drug Administration has approved Wilate for the treatment of spontaneous and trauma-induced bleeding episodes in patients with all types of von Willebrand disease (VWD). Wilate is a newly developed, high-purity, double virus inactivated von Willebrand Factor/Coagulation Factor VIII Concentrate (Human) that demonstrated efficacy for all types of VWD, including pediatric patients, in four prospective clinical trials utilizing both objective and subjective criteria.

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.(1) 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 a 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.

The FDA approval of wilate marks the entrance of Octapharma USA into the U.S. blood coagulation market, with product availability scheduled for early 2010. Octapharma USA is the rapidly growing U.S. division of Octapharma AG, one of the largest plasma products manufacturers in the world.

VWD 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.(2) The illness 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," said 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. Using objective criteria, 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.(3)

"Octapharma's worldwide commitment to coagulation disorders dates back to Octapharma Group's formation 25 years ago," said Octapharma USA President Flemming Nielsen. "We are thrilled that U.S. patients will now have access to wilate following its significant success in Europe as a next generation therapy. Octapharma is committed to providing the U.S. market with life-enhancing therapies."

Since the mid-1980s, the requirements for the viral safety of plasma preparations have constantly been made increasingly stringent, requiring demonstrated virus elimination/inactivation.(4) (5) 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.(6) 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.

Octapharma AG

Headquartered in Lachen, Switzerland, Octapharma is one of the largest plasma products manufacturers in the world and has been committed to patient care and medical innovation for over 25 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 employs over 3,000 people and has biopharmaceutical experience in 80 countries worldwide, including the United States, where Octapharma USA is headquartered in Hoboken, N.J. Octapharma operates two state-of-the-art production sites licensed by the U.S. Food and Drug Administration, providing the highest level of production flexibility and minimizing product shortages. 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.

References

(1) Mollison.P.L., Engelfriet C.P., Contreras M.: Some unfavourable effects of transfusion; in Klein H.G., Anstee D.J. (eds): Mollison's Blood Transfusion in Clinical Medicine. Blackwell Publishing, 2005, pp 666-700.

(2) Centers for Disease Control and Prevention. Bleeding disorders. Available at www.cdc.gov/ncbddd/hbd/hemophilia.htm. Accessed on November 22, 2009.

(3) Wilate Approved Complete Prescribing Information, December 2009.

(4) Note for guidance on virus validation studies: The design, contribution and interpretation of studies validating the inactivation and removal of viruses CPMP/BWP/268/95 February 1996.

(5) Note for guidance on plasma-derived products CPMP/BWP/269/95, rev.2. July 1998.

(6) Farrugia A . Guide for the assessment of clotting factor concentrates for the treatment of Hemophilia. 2003, WFH.

Source: Octapharma USA

CONTACT: Fred Feiner, Yankee Public Relations, fred@yankeepr.com, +1-908-894-3930, for Octapharma

Web Site: http://www.octapharma.com/

Posted: December 2009

Wilate (von Willebrand Factor/Coagulation Factor VIII Complex (Human)) FDA Approval History

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