Octapharma USA: FDA Grants Orphan Drug Exclusivity to wilate®, the First VWF Concentrate for Prophylaxis in All Types of VWD

• Patients with severe VWD are recommended to utilize long-term prophylaxis with VWF concentrate
• FDA provides wilate^® with up to seven years of market exclusivity for approved prophylaxis indication

PARAMUS, N.J., April 29, 2024 /PRNewswire/ -- Octapharma USA, Inc. announced the U.S. Food and Drug Administration (FDA) has granted orphan drug exclusivity for wilate^®, von Willebrand Factor/Coagulation Factor VIII Complex (Human) Lyophilized Powder for Solution for Intravenous Injection, for routine prophylaxis to reduce the frequency of bleeding episodes in adults and children 6 years of age and older with von Willebrand disease (VWD).

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. For additional information, please visit FactorMyWay.com.

About wilate^®
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

Wilate^® is indicated for routine prophylaxis in children 6 years of age and older and adults with von Willebrand disease.

Wilate^® is indicated in adolescents and adults with hemophilia A for:

• Routine prophylaxis to reduce the frequency of bleeding episodes
• On-demand treatment and control of bleeding episodes

CONTRAINDICATIONS
Do not use in patients with known hypersensitivity reactions, including anaphylactic or severe systemic reaction, to human plasma-derived products, any ingredient in the formulation, or components of the container.

WARNINGS AND PRECAUTIONS
Anaphylaxis and severe hypersensitivity reactions are possible. Thromboembolic events may occur. Monitor plasma levels of FVIII activity. Development of neutralizing antibodies to FVIII and to VWF, especially in VWD type 3 patients, may occur. Wilate^® is made from human plasma and carries the risk of transmitting infectious agents.

For full prescribing information, please visit wilateusa.com/pi.

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 octapharmausa.com.

REFERENCES
1 ? U.S. Food & Drug Administration website, Designating an Orphan Product: Drugs and Biological Products, accessed March 29, 2024.

2 ? Robert F. Sidonio, Jr., Ana Boban, Leonid Dubey, Adlette Inati, Csongor Kiss, Toshko Lissitchkov, Dzmitry Novik, Elina Peteva, Ali T. Taher, Margarita Arkadevna Timofeeva, Kateryna V. Vilchevska, Vladimir Vdovin, Sylvia Werner, Sigurd Knaub, Claudia Khayat; Efficacy and Safety of Prophylaxis with a Plasma-Derived Von Willebrand Factor/Factor VIII Concentrate in Previously Treated Patients with Von Willebrand Disease. Blood 2022; 140 (Supplement 1): 8438?8439. doi: https://doi.org/10.1182/blood-2022-162358.

3 ? Centers for Disease Control and Prevention website, What is von Willebrand Disease, accessed March 29, 2024.

SOURCE Octapharma