FDA approves Vonvendi (von Willebrand factor (Recombinant)) to treat von Willebrand Disease- Baxalta

Baxalta Incorporated announced that the FDA has approved Vonvendi [von Willebrand factor (Recombinant)]. Vonvendi is the first and only recombinant treatment for adults living with von Willebrand Disease (VWD). Vonvendi is an innovative recombinant protein treatment that includes a physiologic distribution of proteins called multimers, including ultra-large multimers (ULMs), with large multimers being the most active form of the protein supporting clot formation. The treatment is also the first in the United States that contains only trace amounts of Factor VIII (FVIII), offering the flexibility to administer FVIII only when needed. This attribute allows for tailored treatment for patients who may not require additional FVIII.

VWD is the most common inherited bleeding disorder worldwide, affecting up to one in 100 people; the rarest and often most severe form affects one in 1,000,000 people worldwide. This genetic disorder causes alterations or deficits in von Willebrand factor (VWF), resulting in impaired clotting, and affects women and men equally. The disease can manifest through a variety of bleeding events, including mucosal bleeds, gastrointestinal bleeds or menorrhagia. Patients often live with the disease for years without a proper diagnosis; even with a confirmed diagnosis, there are limited treatment options available.

Comment:The safety and efficacy of Vonvendi were evaluated in two clinical trials of 69 adult participants with VWD. These trials demonstrated that Vonvendi was safe and effective for the on-demand treatment and control of bleeding episodes from a variety of different sites in the body. No safety concerns were identified in the trials. The most common adverse reaction observed was generalized pruritus (itching).