Patients with von Willebrand disease (VWD) now have expanded treatment options, following the FDA’s approval of Vonvendi for prophylactic use in adults aged 18 and older, as well as for managing bleeding episodes and perioperative care in pediatric patients.

Vonvendi was previously authorized solely for on-demand treatment of bleeding episodes & perioperative use in adults with preventive use limited to adults diagnosed with type 3 VWD the most severe type of disease.

The main cause of VWD is low levels of von Willebrand factor (VWF) which leads to error in the clot function of the blood causing excessive bleeding. Vonvendi remains the only recombinant (non–plasma-derived) VWF therapy approved for VWD in the U.S., and this latest decision marks the first approval of a recombinant VWF product for pediatric patients. Until now, treatment options for children were limited exclusively to plasma-derived VWF products.

Clinical studies preformed showed great results regarding Vonvendi’s effectiveness with it’s ability to control bleeding episodes and use in perioperative management in patients with VWD of all ages. It also proved successful for prevention of bleeding episodes in adults with VWD.

In trials, the most common side effects of Vonvendi (≥2% of patients) were headache, nausea, vomiting, dizziness, and itchy skin. Approved under Priority Review with Orphan Drug Designation, the FDA’s decision grants Takeda an important milestone in expanding care for patients with rare bleeding disorders.

Learn more:  FDA Approves Expanded Use of Vonvendi for von Willebrand Disease, Including for Certain Uses for Children