Baxalta Incorporated, a global biopharmaceutical leader, announced that the US Food and Drug Administration (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.

“With no major therapeutic innovation in more than a decade, Vonvendi offers patients an important new option for VWD with a clinical profile that can help them manage this challenging chronic disease,” said Joan Gill, M.D., of the Blood Center and the Medical College of Wisconsin and lead author of the Vonvendi clinical study.

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.

“With its unique feature of ultra-large multimers and the clinical profile seen in our pivotal study, Vonvendi is an important new option with the potential to redefine treatment for adults with von Willebrand disease,” said Brian Goff, executive vice president and president, Hematology, Baxalta. “Vonvendi’s approval represents our commitment to advancing care as we continue to build a strong and differentiated portfolio that can help address challenging bleeding disorders for patients around the world.”

Vonvendi has been approved for on-demand treatment and control of bleeding episodes in adults with von Willebrand disease. The FDA approval was based on positive results from a phase III multicenter, open-label clinical trial that assessed the safety, efficacy and pharmacokinetics of Vonvendi with and without recombinant FVIII. In the pivotal study, all participants (100 percent) reported successful treatment of bleeding episodes, with 96.9 per cent of treated bleeds (N=192 bleeds in 22 patients) achieving an “excellent” efficacy rating and 3.1 per cent achieving a “good” efficacy rating. Most bleeds (81.8 per cent) were resolved with a single infusion, and the treatment showed a mean half-life of 21.9 hours (± 8.36).

No thrombotic events or severe product-related adverse events were observed during the clinical trial, nor were there treatment-related binding or neutralizing antibodies against VWF or neutralizing antibodies against FVIII. The most common adverse reaction observed in greater than two per cent of patients in clinical trials was generalized pruritus.

Baxalta is building a robust clinical development program to optimize patient access to Vonvendi worldwide; a series of clinical programs are planned to evaluate its use for prophylaxis, surgical and paediatric indications. Vonvendi is expected to be broadly available in the United States in late 2016. Baxalta expects to file for regulatory approvals in Europe in 2017 and in other markets around the world.