Octapharma Receives Orphan Drug Exclusivity Approval for wilate(R) - A Replacement Therapy Developed Specifically for von Willebrand Disease

Milestone Marks Swiss Company's Entrance into the U.S. Blood Coagulation Market

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Jan 13, 2010, 10:00 ET

LACHEN, Switzerland, Jan. 13 /PRNewswire/ -- Octapharma AG, one of the largest manufacturers of plasma products in the world, today announced that it has received orphan drug exclusivity approval for wilate® from the U.S. Food and Drug Administration (FDA). The approval was granted for the use of wilate® for the treatment of spontaneous or trauma-induced bleeding episodes in patients with severe von Willebrand disease (VWD) as well as in patients with mild or moderate VWD in whom the use of desmopressin is known or suspected to be ineffective or contraindicated.

The FDA's approval and granting of orphan drug exclusivity 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

"The FDA orphan drug exclusivity approval for wilate® is an important aspect in Octapharma`s development of this drug," said Kim Bjornstrup, Vice Chairman Octapharma Group. "Orphan drug exclusivity confirms Octapharma's decision to focus exclusively on the treatment of von Willebrand patients. Wilate has a combination of two viral attenuation steps, high purity and a physiological 1:1 ratio of VWF and FVIII activity and is approved for treating all VWD patients including those with the most severe, type 3, disease."

About wilate®:

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.

On December 4, 2009, wilate® received FDA approval 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 isolate 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. Wilate® is exclusively derived from large pools of human plasma collected in U.S. FDA approved plasma donation centers and no albumin is added as a stabilizer.

Four prospective clinical trials have demonstrated the safety, tolerability and hemostatic efficacy of wilate® in the treatment of acute bleeding episodes in patients with various types of VWD. Using objective and subjective criteria, treatment with wilate® was observed in 1,068 bleeding episodes and determined to be successful between 84% and 93% with results varying dependent on patient type.(2)

Since the mid-1980s, the requirements for the viral safety of plasma preparations have constantly become more stringent, requiring demonstrated virus elimination/inactivation (3) (4). 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 (5). In the 1980s 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.

Because human plasma contains VWF and FVIII at very low concentration, the wilate® manufacturing process is designed to concentrate the proportion of VWF/FVIII complex while minimizing the inclusion of extraneous proteins. 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.

Important Safety Information

There is a possibility that wilate® can cause hypersensitivity or allergic reactions. Symptoms may include angioedema, burning and stinging at the infusion site, chills, flushing, generalized urticaria, headache, hives, hypotension, lethargy, nausea, restlessness, tachycardia, tightness of the chest, tingling, vomiting, and wheezing. Contact your doctor promptly with any of these symptoms. Let your doctor know if you have had any previous allergic reactions to other VWF/Factor VIII or other plasma products.

The most common related Adverse Reactions observed during the wilate® clinical studies include: urticaria and dizziness. As with all VWF/Factor VIII products, there is the potential for inducing antibodies to Factor VIII and VWF (inhibitors), especially in VWD type 3 patients. Contact your doctor if you are not able to prevent or control bleeding episodes with your regular doses of prescribed VWF/Factor VIII therapy.

There is also a risk of thromboembolic events associated with von Willebrand factor/Coagulation Factor FVIII (VWF/FVIII) products: plasma levels of FVIII activity should be monitored to avoid sustained excessive FVIII levels, which may increase the risk of thrombotic events. There is also a theoretical risk of infectious agents transmission as the product is made from human plasma.

About VWD:

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.(6) The illness is a result of the body's inability to make functional von Willebrand Factor, the human protein that helps clot blood.

About the Octapharma Group:

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 located in Hoboken, N.J. Octapharma operates two state-of-the-art production sites licensed by the U.S. Food and Drug Administration, 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.

References

Wilate® Approved Complete Prescribing Information, December 2009.
Ibid.
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.
Note for guidance on plasma-derived products CPMP/BWP/269/95, rev.2. July 1998.
Farrugia A . Guide for the assessment of clotting factor concentrates for the treatment of Hemophilia. 2003, WFH.
Centers for Disease Control and Prevention. Bleeding disorders. Available at www.cdc.gov/ncbddd/hbd/hemophilia.htm. Accessed on November 22, 2009.

SOURCE Octapharma AG