U.S. FDA Approves Expanded Indication for VONVENDI® [von Willebrand factor (Recombinant)] for Adults and Children with Von Willebrand Disease

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OSAKA, Japan & CAMBRIDGE, Mass.--(BUSINESS WIRE)--Sep 5, 2025--

Takeda (TSE:4502/NYSE:TAK) today announced that the U.S. Food and Drug Administration (FDA) has approved the supplemental Biologics License Application (sBLA) for VONVENDI ® [von Willebrand factor (Recombinant)], expanding the indication to include routine prophylaxis to reduce the frequency of bleeding episodes in adults with von Willebrand Disease (VWD), including those with Type 1 and 2 disease, and on-demand and perioperative management of bleeding in pediatric patients with VWD. VONVENDI was previously approved for on-demand and perioperative use in adults with VWD and routine prophylactic use in adults with severe Type 3 VWD receiving on-demand therapy. 1

VONVENDI is now available for use in a broad range of VWD patients and is the only recombinant von Willebrand Factor (VWF) replacement therapy with approved indications in both adults and children with VWD. 1

VWD is the most common bleeding disorder impacting more than 3 million people in the U.S. alone. It occurs equally among men and women. People with VWD have low levels of or dysfunctional von Willebrand factor (VWF), a protein that helps blood clot. This can lead to life-threatening bleeding – either from an injury or after surgery, gastrointestinal bleeding, dental procedures or childbirth – prolonged nosebleeds, heavy or long menstrual bleeding and easy bruising, which can have a significant impact on quality of life. 2,3

VONVENDI is an infused medicine designed to replace the body's missing or dysfunctional VWF. VONVENDI has a uniquely long half-life (22.6 hours [50 IU/kg] for adults and 14.3 hours for children). In clinical trials, the majority of non-surgical bleeds were treated with one VONVENDI infusion in both adults (157/192) and children (80/104). 1

“The FDA's approval of VONVENDI for routine prophylactic use in adults with all VWD types marks a pivotal milestone in advancing care for this challenging condition. Prophylactic treatment is critical to reduce the frequency of bleeds in adults, including spontaneous and joint bleeds,” said Jonathan C. Roberts, M.D., associate medical and research director at the Bleeding and Clotting Disorders Institute, associate professor of pediatrics and medicine at the University of Illinois College of Medicine at Peoria, and an investigator in the VONVENDI prophylaxis study. “Establishing prophylaxis as the standard of care for all adults with VWD, regardless of subtype, represents a significant step forward in helping to manage bleeding risk and the burdens that bleeds can have on patients’ daily lives.”

This approval is based on data from three clinical trials – a Phase 3 trial in adults with VWD ( NCT02973087 ), a Phase 3 study in children with VWD ( NCT02932618 ) and a Phase 3b continuation trial in adults and children with VWD ( NCT03879135 ) – as well as supportive real world data. 1 Please see the VONVENDI full Prescribing Information for details on the established clinical profile.

“Children and adults living with VWD face unique challenges, including bleeds that can disrupt daily activities like school, work or spending time with loved ones, often leading to physical and emotional burdens,” said Cheryl Schwartz, Senior Vice President, U.S. Rare Disease Business Unit Lead and U.S. Commercial Operations at Takeda. “We are proud to have supported adults with VWD since VONVENDI’s initial approval in 2015 and will now be able to help even more patients manage their condition. This milestone highlights Takeda’s unwavering commitment to the blood and bleeding disorders community as we strive to deliver innovative therapies.”

Takeda submitted applications seeking approval to expand the indications of VONVENDI for on-demand and perioperative management of bleeding in pediatric patients with VWD in Japan and VEYVONDI™ (vonicog alfa) for on-demand use in pediatric patients with VWD in the European Union.

VONVENDI [von Willebrand factor (Recombinant)] Important Information

Indications
VONVENDI [von Willebrand factor (recombinant)] is indicated in adult and pediatric patients with von Willebrand disease (VWD) for:

• On-demand treatment and control of bleeding episodes.
• Perioperative management of bleeding.

For adult patients only:

• Routine prophylaxis to reduce the frequency of bleeding episodes.

Detailed Important Risk Information

CONTRAINDICATIONS
VONVENDI is contraindicated in patients who have had life-threatening hypersensitivity reactions to VONVENDI or constituents of the product (tri-sodium citrate-dihydrate, glycine, mannitol, trehalose-dihydrate, polysorbate 80, and hamster or mouse proteins).

WARNINGS AND PRECAUTIONS
Thromboembolic Events
Thromboembolic events have occurred with VONVENDI. These events can include disseminated intravascular coagulation (DIC), venous thrombosis, pulmonary embolism, myocardial infarction, and stroke. Patients with known risk factors for thrombosis, including low ADAMTS13 levels are at a higher risk. Monitor patients for signs and symptoms of thrombosis such as pain, swelling, discoloration, dyspnea, cough, hemoptysis, and syncope. Institute prophylaxis measures against thromboembolism according to current clinical practice and standard of care.

In patients requiring frequent doses of VONVENDI in combination with rVIII, monitor plasma levels for FVIII:C activity because sustained excessive factor VIII plasma levels can increase the risk of thromboembolic complications.

Hypersensitivity and Infusion-Related Reactions
Hypersensitivity reactions and infusion-related reactions have occurred with VONVENDI. These reactions can include anaphylactic shock, generalized urticaria, angioedema, chest tightness, hypotension, shock, lethargy, nausea, vomiting, paresthesia, pruritus, restlessness, blurred vision, wheezing and /or acute respiratory distress. If signs and symptoms of severe allergic reactions occur, immediately discontinue administration of VONVENDI and provide appropriate supportive care.

VONVENDI contains trace amounts of mouse immunoglobulin G (MuIgG) and hamster proteins less than or equal to 2 ng/IU VONVENDI. Patients treated with this product may develop hypersensitivity reactions to non-human mammalian proteins.

Neutralizing Antibodies
Neutralizing antibodies to VWF and/or factor VIII can occur with VONVENDI. If the expected plasma levels of VWF activity (VWF:RCo) are not attained, perform an appropriate assay to determine if anti-VWF or anti-factor VIII inhibitors are present. Consider other therapeutic options and direct the patient to a physician with experience in the care of either VWD or hemophilia A.

In patients with high levels of inhibitors to VWF or factor VIII, VONVENDI therapy may not be effective, and infusion of this protein may lead to severe hypersensitivity reactions. Since inhibitor antibodies can occur concomitantly with anaphylactic reactions, evaluate patients experiencing an anaphylactic reaction for the presence of inhibitors.

Monitoring Laboratory Tests
Monitor plasma levels of VWF:RCo and factor VIII activities in patients receiving VONVENDI to avoid sustained excessive VWF and/or factor VIII activity levels, which may increase the risk of thrombotic events, particularly in patients with known clinical or laboratory risk factors.

Monitor for development of VWF and/or factor VIII inhibitors when suspected. Perform appropriate inhibitor assays to determine if VWF and/or factor VIII inhibitors are present if bleeding is not controlled with the expected dose of VONVENDI.

ADVERSE REACTIONS
The most common adverse reactions observed in greater than or equal to 2% of patients in clinical trials with VONVENDI (n=132) were headache, vomiting, nausea, dizziness, and generalized pruritus.

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. 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. 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. Bleeding caused by VWD varies greatly among patients with this disease. 2

About Takeda
Takeda is focused on creating better health for people and a brighter future for the world. We aim to discover and deliver life-transforming treatments in our core therapeutic and business areas, including gastrointestinal and inflammation, rare diseases, plasma-derived therapies, oncology, neuroscience and vaccines. Together with our partners, we aim to improve the patient experience and advance a new frontier of treatment options through our dynamic and diverse pipeline. As a leading values-based, R&D-driven biopharmaceutical company headquartered in Japan, we are guided by our commitment to patients, our people and the planet. Our employees in approximately 80 countries and regions are driven by our purpose and are grounded in the values that have defined us for more than two centuries. For more information, visit www.takeda.com.

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Takeda Medical Information
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References:

1. VONVENDI [von Willebrand factor (recombinant)] Prescribing Information; 2025. https:/www.shirecontent.com/PI/PDFs/VONVENDI_USA_ENG.pdf
2. What is von Willebrand disease? Centers for Disease Control and Prevention website. Accessed July 9, 2025. https://www.cdc.gov/von-willebrand/about/?CDC_AAref_Val=https://www.cdc.gov/ncbddd/vwd/facts.html
3. Nicholas L.J. Chornenki, Edwin Ocran, Paula D. James; Special considerations in GI bleeding in VWD patients. Hematology Am Soc Hematol Educ Program 2022; 2022 (1): 624–630. doi: https://doi.org/10.1182/hematology.2022000390

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PUB: 09/05/2025 08:45 PM/DISC: 09/05/2025 08:44 PM

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