FDA Approves Expanded Use of Vonvendi for VWD in Adults and Children

The FDA approved expanded use of von Willebrand factor, recombinant (Vonvendi; Takeda Pharmaceuticals), for routine preventative use in adults aged 18 years and older for all types of von Willebrand disease (VWD), along with treatment of bleeding episodes and perioperative use in children with VWD.^1,2

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“Today’s approval is a testament to the collaborative interactions between CBER review teams and product developers, aimed at making innovative therapies available for use in pediatric populations at the same time as adults,” Vijay Kumar, MD, acting director of the Center for Biologics Evaluation and Research (CBER) Office of Therapeutic Products, said in a news release.¹

Understanding Von Willebrand Disease

VWD is a bleeding disorder in which an individual’s blood does not clot properly, caused by low levels of von Willebrand factor (VWF)—a protein that helps blood clot—or the protein does not react as it should. Most individuals with the disease are born with it by inheriting it from 1 or both parents; however, signs and symptoms may not occur for years. This includes excessive bleeding from an injury or after surgery or dental work; frequent nosebleeds that do not stop within 10 minutes; heavy or long menstrual bleeding; heavy bleeding during labor or delivery; blood in the urine or stool; and easy bruising or lumpy bruises. Although VWD cannot be cured, treatment and self-care can help individuals manage the disease and its symptoms.³

“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,” Cheryl Schwartz, senior vice president, US rare disease business unit lead, and US commercial operations at Takeda, said in a news release.²

Recombinant Von Willebrand Factor for Von Willebrand Disease

The drug is an infused medication that works by replacing the body’s missing or defective VWF. It is marked as the only recombinant, or nonplasma-derived, VWF product granted for VWD that received FDA approval in 2015 and is also the first recombinant VWF product that has been approved for pediatric patients in the US. The infusion has a long half-life, lasting about 22.6 hours in adults and 14.3 hours in children.^1,2

The approval is based on positive data from 3 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).^4-6

Results from the trials demonstrated successful treatment control of bleeding episodes and the use of perioperative management in patients with VWD of all ages, along with success for prevention of bleeding episodes in adults with VWD.¹

“This approval demonstrates FDA flexibility in evaluating applications of therapeutics to treat rare diseases,” Vinay Prasad, MD, MPH, director of the FDA’s CBER, said in the news release. “When we see the trifecta of plausible mechanism of action, robust pharmacology/biologic science, and supportive clinical study data, we promptly act even if that data is derived from a small sample size study.”¹

The most common adverse reactions included headache, vomiting, nausea, dizziness, and itchy skin, which were reported to occur in about 2% of patients.¹

“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,” Jonathan C. Roberts, MD, 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 [von Willebrand factor, recombinant] prophylaxis study, said in the news release.²

REFERENCES

1. FDA Approves Expanded Use of Vonvendi for von Willebrand Disease, Including for Certain Uses for Children. FDA. News release. September 5, 2025. Accessed September 8, 2025. https://www.fda.gov/news-events/press-announcements/fda-approves-expanded-use-vonvendi-von-willebrand-disease-including-certain-uses-children

2. U.S. FDA Approves Expanded Indication for VONVENDI® [von Willebrand factor (Recombinant)] for Adults and Children with Von Willebrand Disease. Takeda. News release. September 5, 2025. Accessed September 8, 2025. https://www.takeda.com/newsroom/newsreleases/2025/expanded-indication-vonvendi/

3. Von Willebrand disease. Mayo Clinic. News release. October 26, 2021. Accessed September 8, 2025. https://www.mayoclinic.org/diseases-conditions/von-willebrand-disease/symptoms-causes/syc-20354978

4. RVWF IN PROPHYLAXIS. National Library of Medicine. Updated August 6, 2021. Accessed September 8, 2025. https://clinicaltrials.gov/study/NCT02973087

5. A Study of Recombinant Von Willebrand Factor (rVWF) With or Without ADVATE in Children With Severe Von Willebrand Disease (VWD). National Library of Medicine. Updated April 30, 2025. Accessed September 8, 2025. https://clinicaltrials.gov/study/NCT02932618

6. A Study of Recombinant Von Willebrand Factor (rVWF) in Pediatric and Adult Participants With Severe Von Willebrand Disease (VWD). National Library of Medicine. Updated September 3, 2025. https://clinicaltrials.gov/study/NCT03879135