The first recombinant von Willebrand factor has been approved for von Willebrand disease, the Food and Drug Administration announced.
Dr. Karen Midthun, director of the FDA’s Center for Biologics Evaluation and Research, said in the Dec. 8 statement that the approval “provides an additional therapeutic option for the treatment of bleeding episodes in patients with von Willebrand disease.”
The product was approved for on-demand therapy for bleeding episodes in adults with the disorder, which affects up to 1% of the U.S. population. Current treatment options include desmopressin and plasma-derived von Willebrand factor/factor VIII concentrates. The latter are limited by donor availability and are variable in the factor VIII levels they contain.
The new treatment, marketed as Vonvendi (Baxalta), is administered by infusion. The product contains only trace amounts of factor VIII, allowing for administration of factor VIII only as needed.
In a phase III manufacturer-sponsored nonrandomized trial of 22 subjects, bleeding was controlled with a single infusion in 82% of 192 bleeding episodes, according to a Baxalta press release. Patients with major bleeding required as many as four infusions, but treatment was effective in all subjects and 97% had an excellent efficacy rating and the remaining 3% had a good efficacy rating.
The most common adverse event was pruritus. No thrombotic events or severe allergic reactions were reported. Patients did not develop neutralizing antibodies against von Willebrand factor or factor VIII during the course of the study.
In its news release, the manufacturer said it is building a clinical development program to increase patient access to the treatment and evaluate its use in “prophylaxis, surgical, and pediatric indications.” Vonvendi is expected to be available in late 2016.