Octapharma receives Orphan Drug Exclusivity Approval for wilate(R) - the First Replacement Therapy Developed Specifically for von Willebrand Disease

LACHEN--13 Jan--PRNewswire-AsiaNet/InfoQuest

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

Octapharma AG, one of the largest plasma products manufacturers in the world, today announced that it has received orphan exclusivity approval for wilate(R) from the U.S. Food and Drug Administration (FDA). The designation was granted for the use of wilate(R) 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 approval and granting of orphan drug exclusivity of wilate(R) 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.

"The FDA orphan drug exclusivity approval for wilate(R) is an important aspect in Octapharma's development of this drug," said Kim Bjornstrup, Vice Chairman Octapharma Group. "One requirement to receive orphan drug exclusivity was to present to the FDA an explanation of why wilate(R) may be clinically superior to existing therapies (1). Wilate(R) is specifically developed and manufactured for the treatment of VWD. Its unique combination of two viral attenuation steps, high purity and a physiological 1:1 ratio of FVIII

and VWF will provide a next generation treatment option for patients with von Willebrand disease."

About wilate(R):

Wilate(R) 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(R) received FDA registration 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. (2) wilate(R) 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(R) 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(R) in the treatment of acute bleeding episodes and prophylaxis in patients with various types of VWD.

Using objective criteria, wilate(R) 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)

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(R) 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(R) manufacturing contributes to the viral safety.

Human plasma contains VWF and FVIII at very low concentration. The wilate(R) 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.

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.(7) 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(R) (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 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) Department of Health and Human Services Food and Drug Administration 21 CFR Part 316 (Docket No. 85N-0483), RIN 0905-AB55 Orphan Drug regulations, Subpart C Available at

www.fda.gov/ForIndustry/DevelopingProductsforRareDiseasesConditions/HowtoapplyforOrphanProductDesignation/ucm135122.htm

(2) Wilate(R) Approved Complete Prescribing Information, December 2009.

(3) Ibid.

(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.

(7) 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

CONTACT: Olaf Walter, MD, PhD

Head of International Business Unit Coagulation

[email protected]

+41-55-4512-115

--Distributed by AsiaNet ( www.asianetnews.net )--

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