MEDIA RELEASE PR37808
Octapharma receives Orphan Drug Exclusivity Approval for wilate(R) - the First Replacement Therapy
Developed Specifically for von Willebrand Disease
LACHEN, Jan. 13 / PRNewswire-AsiaNet/ --
- 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
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
nation/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.
November 22, 2009.
THIS PRESS RELEASE IS NOT INTENDED FOR PUBLICATION IN THE U.S.
SOURCE: Octapharma AG
CONTACT: Olaf Walter, MD, PhD
Head of International Business Unit Coagulation
olaf.walter@octapharma.ch
+41-55-4512-115
Translations:
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