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
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.
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
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
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
(2) Wilate(R) Approved Complete Prescribing Information, December 2009.
(3) Ibid.
(4) Note for guidance on virus validation studies: The design,
(5) Note for guidance on plasma-derived products CPMP/BWP/269/95, rev.2.
(6) Farrugia A . Guide for the assessment of clotting factor concentrates
(7) Centers for Disease Control and Prevention. Bleeding disorders.
SOURCE: Octapharma AG
CONTACT: Olaf Walter, MD, PhD
Head of International Business Unit Coagulation
+41-55-4512-115
--Distributed by AsiaNet ( www.asianetnews.net )--
Octapharma announced today that the final results from the NuProtect study on the immunogenicity of Nuwiq(R) in previously untreated patients (PUPs) with severe haemophilia A have been published in the leading medical journal Thrombosis and Haemostasis (Liesner RJ et al. "Simoctocog Alfa (Nuwiq(R)) in Previously Untreated Patients with Severe Haemophilia A: Final Results of the NuProtect Study" https://www.thieme-connect.com/products/ejournals/abstract/10.1055/s-0040-1722623). The NuProtect