- Shire on Tuesday launched Vonvendi in the U.S., bringing the first recombinant von Willebrand factor treatment to market eight months after winning approval from the Food and Drug Administration.
- Von Willebrand disease is a hereditary bleeding disorder affecting an estimated 1 in 100 people, or roughly 3 million people in the U.S., according to the company. VWD is often difficult to diagnose, however, and the missing von Willebrand fractor (rVWF) leads patients to experience spontaneous bleeds.
- Notably, Vonvendi allows for treatment without the use of Factor VIII, a standard treatment for hemophilia A but not specifically designed to treat VWD.
When Baxalta announced the approval of Vonvendi last December, it was the second approval in the space of one month for the company, which was still attempting to shake off Shire's pursuit. But Shire prevailed and bought out Baxalta for $32 billion in January 2016.
Now Shire is launching Vonvendi, touting it as the 'first and only' rVWF treatment for patients with VWD. This positioning matters because until now, hematologists have had to treat patients with VWD with Factor VIII, a treatment intended for treatment of hemophilia A.
"The ability to dose VWF and FVIII separately is important in this disorder; not every patient needs the additional FVIII present in conventional plasma-derived VWF products, and using these products may require additional careful monitoring," said Jeff Schaffnit, head of U.S. hematology for Shire, in an email to Biopharma Dive.
Results from the 37-patient Phase 3 study showed one infusion of Vonvendi was sufficient to manage over 80% of bleeds, and all patients reported successful management of bleeding episodes.
In December, financial analyst firm ThomsonOne predicted potential sales of $400 million to $500 million for Vonvendi. However, Shire hopes to broaden the label which could help boost sales further.
"We’re actively investigating the potential use of Vonvendi in additional indications including pediatrics, surgery and prophylaxis as we work to deliver new solutions to people around the world who suffer from bleeding disorders," Schaffnit said.