Baxalta, a global biopharmaceutical provider dedicated to delivering transformative therapies to patients with orphan diseases and underserved conditions, has announced that the FDA has approved VONVENDI, for treatment of von Willebrand disease (VWD).

VONVENDI is the first and only recombinant treatment for adults living with the disease.

VONVENDI is a 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 U.S. that contains only trace amounts of Factor VIII (FVIII), offering the flexibility to administer FVIII only when needed. That 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 million people worldwide. The 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.

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 reported successful treatment of bleeding episodes, with 96.9% of treated bleeds (N=192 bleeds in 22 patients) achieving an “excellent” efficacy rating and 3.1% achieving a “good” efficacy rating. Most bleeds (81.8%) were resolved with a single infusion, and the treatment showed a mean half-life of 21.9 hours (± 8.36).

Baxalta is building a 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 pediatric indications.

VONVENDI is expected to be broadly available in the U.S. in late 2016. Baxalta expects to file for regulatory approvals in Europe in 2017 and in other markets around the world.