The Food and Drug Administration (FDA) has approved Vonvendi® (von Willebrand factor [recombinant]) for routine prophylaxis to reduce the frequency of bleeding episodes in adults 18 years of age and older with severe Type 3 von Willebrand disease (VWD) receiving on-demand therapy.
The approval was based on data from a prospective, open-label, nonrandomized, multicenter, phase 3 study (ClinicalTrials.gov Identifier: NCT02973087) that assessed the efficacy and safety of prophylactic treatment with Vonvendi in reducing the frequency of bleeding episodes in adults with severe Type 3 VWD (n=10) over a period of 12 months. The primary endpoint was the median annualized bleeding rate (ABR) for all bleeds, including spontaneous and joint bleeds.
Results showed the median ABR for all bleeds was reduced from a historical median ABR of 5.0 (range, 3.0-159.0) to an on-study median ABR of 2.3 (range, 0-157.9), corresponding to a 54.7% reduction. Vonvendi also reduced the median ABR for spontaneous bleeds by 75.9% and joint bleeds by 100%. The most common adverse reactions reported with Vonvendi included headache, vomiting, nausea, dizziness, arthralgia, joint injury, vertigo, increased alanine transaminase, and generalized pruritus.
“This approval is a major advancement for those living with severe Type 3 VWD and is a testament to Takeda’s commitment to improve VWD care,” said Heather Dean, Vice President, US Hematology Franchise Head, Takeda. “With routine prophylactic treatment, there is now a proactive strategy available for management of bleeding episodes and may offer people living with severe Type 3 VWD hope that bleed reduction is possible.”
Vonvendi, a recombinant von Willebrand factor, is also indicated for use in adults with VWD for on-demand treatment and control of bleeding episodes, and for perioperative management of bleeding.
The product is available as a lyophilized powder in single-dose vials containing nominally 650 or 1300 international units VWF:RCo.
- FDA approves prophylactic treatment with Vonvendi® [von willebrand factor (recombinant)] for adult patients living with severe type 3 von Willebrand disease (VWD). News release. Takeda Pharmaceutical Company Limited. Accessed January 31, 2022. https://www.businesswire.com/news/home/20220131005214/en/FDA-Approves-Prophylactic-Treatment-with-VONVENDI%C2%AE-von-Willebrand-Factor-Recombinant-for-Adult-Patients-Living-with-Severe-Type-3-von-Willebrand-Disease-VWD
- Vonvendi. Package insert. Takeda Pharmaceutical Company Limited; 2021. Accessed January 31, 2022. https://www.shirecontent.com/PI/PDFs/VONVENDI_USA_ENG.pdf