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

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 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

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.

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.

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.

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.

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.