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U.S. Food and Drug Administration Accepts Takeda’s Supplemental Biologics License Application for Adult Prophylactic Use of VONVENDI® [von Willebrand factor (Recombinant)]

June 14, 2021
  • If approved VONVENDI would be the first replacement therapy for prophylactic use in adults with von Willebrand disease (VWD) in the U.S.
  • VWD is the most common inherited bleeding disorder, affecting up to one percent of the U.S. population1

Cambridge, Mass. June 14, 2021 – Takeda Pharmaceutical Company Limited (TSE:4502/NYSE:TAK) (“Takeda”) announced today that the United States Food & Drug Administration (FDA) has accepted for review the supplemental Biologics License Application (sBLA) for VONVENDI® [von Willebrand factor (Recombinant)] for prophylactic treatment to prevent or reduce the frequency of bleeding episodes in adults (age 18 and older) with von Willebrand disease (VWD). The FDA has provided an anticipated Prescription Drug User Fee Act (PDUFA) action date of January 28, 2022.

VONVENDI is currently indicated for use in adults with VWD for on-demand treatment and control of bleeding episodes and perioperative management of bleeding. VONVENDI is the only recombinant von Willebrand factor (VWF) replacement therapy. It is an infused product that is specifically designed to replace the body's missing or dysfunctional VWF, which is needed to form clots as well as carry and protect coagulation Factor VIII from rapid elimination.

“Many people living with von Willebrand disease are in need of additional therapeutic options.” said Heather Dean, Vice President, U.S. Hematology Franchise Head. “We are excited about this important milestone, as it supports our commitment to bring innovative products to patients together with personalized support to the bleeding disorder community.”

The application is based on data from a prospective, open-label, non-randomized, multicenter, phase 3 study in adult patients with severe VWD over a period of 12 months. The results of this phase 3 study will be presented at the International Society of Thrombosis and Haemostasis (ISTH) Congress on July 20th, 2021.

VONVENDI [von Willebrand factor (recombinant)] Important Information

What is VONVENDI?

VONVENDI is used in adults (age 18 years and older) diagnosed with von Willebrand disease to:

  • treat and control bleeding episodes
  • prevent excessive bleeding during and after surgery
Detailed Important Risk Information
Who should not use VONVENDI?

You should not use VONVENDI if you:

  • Are allergic to any ingredients in VONVENDI.
  • Are allergic to mice or hamsters.

Tell your healthcare provider if you are pregnant or breastfeeding because VONVENDI may not be right for you.

How should I use VONVENDI?

Your first dose of VONVENDI for each bleeding episode may be administered with a recombinant factor VIII as instructed by your healthcare provider.

Your healthcare provider will instruct you whether additional doses of VONVENDI with or without recombinant factor VIII are needed.

What should I tell my healthcare provider before I use VONVENDI?

You should tell your healthcare provider if you:

  • Have or have had any medical problems.
  • Take any medicines, including prescription and non-prescription medicines, such as over-the-counter medicines, supplements or herbal remedies.
  • Have any allergies, including allergies to mice or hamsters.
  • Are breastfeeding. It is not known if VONVENDI passes into your milk and if it can harm your baby.
  • Are pregnant or planning to become pregnan It is not known if VONVENDI can harm your unborn baby.
  • Have been told that you have inhibitors to von Willebrand factor (because VONVENDI may not work for you).
  • Have been told that you have inhibitors to blood coagulation factor VIII.
What else should I know about VONVENDI and von Willebrand Disease?

Your body can form inhibitors to von Willebrand factor or factor VIII. An inhibitor is part of the body’s normal defense system. If you form inhibitors, it may stop VONVENDI or factor VIII from working properly. Consult with your healthcare provider to make sure you are carefully monitored with blood tests for the development of inhibitors to von Willebrand factor or factor VIII.

What are the possible side effects of VONVENDI?

You can have an allergic reaction to VONVENDI.

Call your healthcare provider right away and stop treatment if you get a rash or hives, itching, tightness of the throat, chest pain or tightness, difficulty breathing, lightheadedness, dizziness, nausea or fainting.

Side effects that have been reported with VONVENDI include: nausea, vomiting, tingling or burning at infusion site, chest discomfort, dizziness, hot flashes, itching, high blood pressure, muscle twitching, unusual taste, blood clots and increased heart rate.

Tell your healthcare provider about any side effects that bother you or do not go away.

You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch, or call 1-800-FDA-1088.

Please see VONVENDI full Prescribing Information.

About von Willebrand disease (VWD)

VWD is the most common inherited bleeding disorder, affecting up to one percent of the U.S. population.1 VWD is caused by a deficiency or dysfunction of von Willebrand factor (VWF), one of several types of proteins in the blood that are needed to facilitate proper blood clotting.1 Due to this defect or deficiency in VWF, blood is not able to clot effectively in people with VWD, which may result in heavy menstrual periods, easy bruising or frequent nose bleeds.1 Bleeding caused by VWD varies greatly among patients with this disease.2


References

  1. National Hemophilia Foundation. "Von Willebrand Disease." National Hemophilia Foundation website. https://www.hemophilia.org/Bleeding-Disorders/Types-of-Bleeding-Disorders/Von-Willebrand-Disease. Last accessed June 2021.
  2. National Hemophilia Foundation. "VWD Summit Highlights." HEMAWARE website. https://hemaware.org/story/vwd-summit-highights.
About Takeda Hematology

Following its recent acquisition of Shire, Takeda is a leader in hemophilia with the longest heritage and market-leading portfolio, backed by established safety and efficacy profiles with decades of real-world experience. We have 70+ years driving innovation for patients and a broad portfolio of 11 products across multiple bleeding disorders. Our experience as leaders in hematology means we are well prepared to meet today’s needs as we pursue future developments in the care of bleeding disorders. Together with the hematology community, we are raising expectations for the future, including earlier diagnosis, earlier and full protection against bleeds, and more personalized patient care.

About Takeda Pharmaceutical Company Limited

Takeda Pharmaceutical Company Limited (TSE:4502/NYSE:TAK) is a global, values-based, R&D-driven biopharmaceutical leader headquartered in Japan, committed to discover and deliver life-transforming treatments, guided by our commitment to patients, our people and the planet. Takeda focuses its R&D efforts on four therapeutic areas: Oncology, Rare Genetic and Hematology, Neuroscience, and Gastroenterology (GI). We also make targeted R&D investments in Plasma-Derived Therapies and Vaccines. We are focusing on developing highly innovative medicines that contribute to making a difference in people's lives by advancing the frontier of new treatment options and leveraging our enhanced collaborative R&D engine and capabilities to create a robust, modality-diverse pipeline. Our employees are committed to improving quality of life for patients and to working with our partners in health care in approximately 80 countries. For more information, visit https://www.takeda.com.

For more information, visit https://www.takeda.com.

Media Contact:
David Murdoch
david.murdoch@takeda.com
+1 781-421-1741              

 

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