Von Willebrand disease drugs approved

Two Medications Approved For Von Willebrand Disease, Menorrhagia

New drugs could be available in 2010
Author: Martha Frase

In late 2009, the US Food and Drug Administration (FDA) approved two new medications of interest to the bleeding disorders community: Lysteda™, used to treat heavy menstrual bleeding common in women with von Willebrand disease (VWD), and Wilate™, for bleeding in all types of VWD.

Lysteda is convenient because it is taken orally in tablet form. The drug helps to stabilize the blood-clotting protein fibrin, preventing it from breaking down and causing excessive bleeding. It is made by Xanodyne Pharmaceuticals of Newport, Kentucky.

Lysteda is tranexamic acid in a sustained slower-release formulation, which allows for less frequent dosing than the standard-dose version. Tranexamic acid, in a standard-dose formulation, has been used in other countries since the mid-1980s, but in the US had been approved only as an injectable to reduce or prevent bleeding after tooth extraction in people with hemophilia.

“In many parts of the world it is one of the first-line treatments for heavy menstrual bleeding, but the manufacturers have not made it available here, which has been a travesty for US patients,” says Peter A. Kouides, MD, medical and research director at the Mary M. Gooley Hemophilia Center in Rochester, New York.

Kouides, who has been involved in researching the efficacy of standard dose tranexamic acid, noted that the drug is about five times more potent than Amicar™, which is now available in the US.

In an April 2009 study published in the British Journal of Haematology, Kouides and colleagues at the Centers for Disease Control and Prevention showed that standard dose tranexamic acid reduced menstrual blood flow and improved quality of life in women with menorrhagia and an underlying bleeding disorder. It was compared with intranasal DDAVP (Stimate®). Although Stimate also showed benefit, improvement was statistically more significant with the tranexamic acid. Kouides says the drug has few marked side effects.

Another VWD Option

Wilate is a newly developed von Willebrand factor/coagulation factor VIII concentrate to treat spontaneous and trauma-induced bleeding episodes in people with severe VWD and in those with mild or moderate VWD when desmopressin is ineffective. Wilate is made by Swiss pharmaceutical manufacturer Octapharma.

“The FDA approval of Wilate makes this the first and only replacement therapy developed and manufactured specifically for VWD,” according to Craig Kessler, MD, professor of medicine and pathology and director of the Division of Coagulation at Georgetown University Hospital in Washington, DC. He is also chair of the National Hemophilia Foundation’s Medical and Scientific Advisory Council (MASAC). “Wilate is a next-generation treatment option for patients with von Willebrand disease.”

Wilate joins Humate P™ and Alphanate™ as options for people with VWD who need to infuse.

“The more pharmaceutical companies supplying products enriched with VWD factor, the better for the caregiver and the patient,” Kouides says. “With this addition, it is nice to know there are unlikely to be supply issues for people who need it.”

Although both drugs received FDA approval late in 2009, they were not yet available as of spring 2010. Once drugs are approved, it takes time to meet the demand.

“Check with your hematologist or local hemophilia center,” Kouides advises patients.