The FDA Orphan Drug Designation program provides orphan status to biologics and drugs for rare diseases. Wilate® is the first von Willebrand factor (VWF) concentrate indicated for prophylactic treatment across all forms of VWD, a significant patient treatment milestone for the most prevalent bleeding disorder in the United States. VWD affects up to 1% of the U.S. population, including many who are undiagnosed.
"The FDA orphan exclusivity is exciting news for Octapharma and patients who have endured excessive bleeding episodes," said Octapharma USA President Flemming Nielsen. "Patients with severe VWD are recommended to utilize long-term prophylaxis with VWF concentrate, as compared to on-demand treatment for bleeding, to avoid life-threatening bleeding episodes as well as a better quality of life."
FDA orphan drug designation provides pharmaceutical manufacturers with the potential for seven years of market exclusivity, tax credits for qualified clinical trials, and exemption from user fees.
A Life-Saving Treatment Option for Many Patients
"VWF prophylaxis may be life-saving and life-altering for many patients, and lead to improved patient care and a reduced burden of disease for the healthcare system," said Akshat Jain, MD, Director of the Inherited Bleeding Disorder and Thrombosis Program at Loma Linda University School of Medicine and Children's Hospital. "The orphan drug designation is another example of Octapharma's impressive support of the bleeding disorders community."
Factor My Way: Empowering VWD Patients:
The Factor My Way patient support program is designed by Octapharma USA for those living with VWD and hemophilia A. The free membership program provides access to caregivers, educational programs, a resource library, and more.
Wilate®, von Willebrand Factor/Coagulation Factor VIII Complex (Human) Lyophilized Powder for Solution for Intravenous Injection is indicated in children and adults with von Willebrand disease for:
On-demand treatment and control of bleeding episodes
Perioperative management of bleeding
Routine prophylaxis to reduce the frequency of bleeding episodes
About Octapharma:
Headquartered in Lachen, Switzerland, Octapharma is one of the largest human protein manufacturers in the world, developing and producing human proteins from human plasma and human cell lines.
Octapharma employs nearly 12,000 employees worldwide to support the treatment of patients in 118 countries with products across three therapeutic areas: Immunotherapy, Hematology and Critical Care.
Octapharma has seven R&D sites and five state-of-the-art manufacturing facilities in Austria, France, Germany and Sweden, and operates more than 190 plasma donation centers across Europe and the US. Octapharma has 40 years of experience in patient care. The company's American subsidiary, Octapharma USA, is located in Paramus, N.J.
For more information, please visit www.octapharmausa.com.
