A treatment for an inherited bleeding disorder has been approved by the US Food and Drug Administration (FDA).
Von Willebrand disease (VWD) affects around 1% of the US population and is caused by a deficiency or defect in a protein critical for normal blood clotting called von Willebrand factor.
The treatment, recombinant von Willebrand factor (Vonvendi; Baxalta), was approved for on-demand treatment and control of bleeding episodes in adults aged 18 years and older with the disease.
Patients with VWD can develop severe bleeding from the nose, gums and intestines, and into muscles and joints. Affected women can experience heavy menstrual periods and excessive bleeding after childbirth.
“The approval of Vonvendi provides an additional therapeutic option for the treatment of bleeding episodes in patients with VWD,” says Karen Midthun, director of the FDA’s Center for Biologics Evaluation and Research.
Two clinical trials involving 69 adult participants with VWD showed the treatment was safe and effective for on-demand treatment and control of bleeding from different sites in the body, and there were no safety concerns identified. The most common adverse reaction was generalised pruritus (itching).
The FDA had granted the treatment orphan product designation, which is used to promote the development of drugs intended to treat rare diseases.