FDA Approves von Willebrand Factor (Recombinant) as Prophylaxis for Adults with Severe Type 3 Disease

The FDA has approved von Willebrand factor (recombinant) for routine prophylaxis to reduce the frequency of bleeding episodes in patients with severe type 3 von Willebrand disease (VWD) who are receiving on-demand therapy, according to a Takeda Pharmaceuticals press release.

The treatment is the only recombinant von Willebrand factor (VWF) replacement therapy and is the first and only FDA-approved treatment to reduce the frequency of bleeding episodes for severe type 3 VWD with routine prophylactic use, according to Takeda. It is also indicated for adults with severe type 3 VWD who are receiving on-demand and perioperative bleeding management.

VWD is an inherited disorder caused by a deficiency or defective function of VWF, which is one of several types of proteins in the blood that are necessary for proper clotting. Because of this defective function or deficiency, blood is not able to clot effectively in individuals with the disease, which can impact men and women equally. Von Willebrand factor (recombinant) is an infused product that is designed to replace the missing or dysfunctional VWF, according to the press release.

“Von Willebrand disease is a complex disease where both patients and providers may experience stress and uncertainty due to the unpredictable disease course and limited treatment options,” said Miguel A. Escobar, MD, an investigator in the VONVENDI prophylaxis study, in the press release. “A prophylactic treatment option may allow for greater disease control and the potential to enhance the standard of care.”

The approval was based on data from a prospective, open-label, international multicenter study to evaluate the efficacy and safety of prophylactic treatment in reducing the frequency of bleeding episodes for 10 adult patients diagnosed with severe type 3 VWD who were previously treated on-demand. Based on descriptive statistics, the median annualized bleeding rates for all bleeds was reduced from a historical median of 5 to an on-study median of 2.3, which is a 54.7% reduction.

Furthermore, the most common adverse reactions occurring in patients treated with von Willebrand factor (recombinant) were headache, vomiting, nausea, dizziness, arthralgia, joint injury, vertigo, increased alanine transaminase, and generalized pruritus.

“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 von Willebrand disease hope that bleed reduction is possible,” said Heather Dean, vice president and US hematology franchise head at Takeda, in the press release.

von Willebrand Factor (Recombinant)

A recombinant von Willebrand factor (rVWF) was recently approved in the United States for on-demand treatment and control of bleeding episodes in adults with von Willebrand disease (VWD). In contrast to plasma-derived VWF products available in the United States, rVWF does not contain factor VIII (FVIII).

An infusion of 1 IU/kg of vWF:Rco is said to raise the plasma level of vWF:Rco by around 1.5 IU/dL. In general, an initial dose of plasma vWF concentrate of 40 to 60 international units (expressed as units of ristocetin cofactor activity [vWF:RCo]) per kg/body weight is given to obtain plasma levels of 50 to 100 IU/dL