Veyvondi effective, reduces costs, as VWF replacement therapy: Study
Lab-made treatment found to match plasma-derived concentrates
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A real-world study has found that during hospitalizations among people with von Willebrand disease (VWD), VWF replacement therapy with lab-made Veyvondi (vonicog alfa) is as effective as using plasma-derived VWF (pdVWF) concentrates.
Veyvondi (marketed as Vonvendi in the U.S.) was also less expensive and used less often than pdVWF, particularly among those with VWD type 1.
Those are among the results of the Hopscotch-Will 2 study (NCT04887324), which compared the two VWF replacement therapy versions in adults with VWD treated in hospitals in France over a four-year span.
“The data collected … indicate that the different products are equivalent,” the researchers wrote, noting that “[Veyvondi] was used in similar patient profiles and for identical procedures.”
However, the team noted, “the cost of treatment with [Veyvondi] was significantly lower.”
The study, “Vonicog Alfa versus Plasma-Derived von Willebrand Factor During Hospitalization: Results of an Observational Retrospective Multicenter Study,” was published in the journal Seminars in Thrombosis and Hemostasis by scientists in France.
A bleeding disorder, VWD is caused by a deficiency or dysfunction of von Willebrand factor (VWF), a protein that’s essential for blood clotting. Without enough VWF, the body cannot properly form blood clots, leading to abnormally heavy and prolonged bleeding.
People with milder forms of VWD are often treated with medicines such as tranexamic acid and desmopressin, which help control smaller bleeds or prevent bleeding during minor procedures. Those with more severe VWD often need VWF replacement therapy, which provides a functional form of the missing protein. These treatments are used for serious bleeding and can also be given regularly to prevent bleeding in severe cases.
Testing lab-made vs. plasma-based VWF replacement therapy
There are two main types of VWF replacement therapy used among patients. One is a lab-made version called Veyvondi. The other type is made from human plasma — the liquid portion of blood without cells — and includes products such as Alphanate, Humate-P, and Wilate. These are called plasma-derived VWF (pdVWF) concentrates.
In 2023, a team led by researchers at Nantes Université in Western France published results from the first Hopscotch-Will study (NCT03875924). That study examined the use of plasma-derived VWF across five French hospitals. It found that younger patients and more invasive procedures were associated with greater use of pdVWF.
The research team has now published results from Hopscotch-Will 2, which followed adults with VWD treated in French hospitals from January 2019 to December 2022. This work specifically compared the use of pdVWF and Veyvondi.
A total of 866 adults with VWD types 1 or 2 were involved in the study. Among them, 285 patients had 648 hospitalizations. During those hospital stays, 126 patients received VWF treatment during 249 hospital stays.
Reasons for these hospitalizations included tests of the digestive system, childbirth and in vitro fertilization, and dental injuries. Patients were also hospitalized for trauma, mostly for major surgeries.
Active bleeding was involved in about one-fourth of hospitalizations (26%). Among these, melena, or black, tarry stools caused by internal bleeding, was present in nearly half (48%) of patients.
VWD type 1 patients given Veyvondi needed a lot less
When the researchers compared treatments, Veyvondi was used more often than pdVWF during hospital stays (61% vs. 39%). Specifically, 73 patients received Veyvondi alone, and 16 patients received it with factor VIII (FVIII). In comparison, 39 patients received combined pdVWF/FVIII, 21 patients received pdVWF alone, and two patients received pdVWF and FVIII as separate treatments.
The study also found that people with VWD type 1 needed significantly less Veyvondi than pdVWF when measured as IU/kg per patient per year. However, this difference was not seen in patients with VWD type 2.
Across the main medical areas — digestive care, dental, gynecology/obstetrics, and trauma care — there was no meaningful difference in the amount of treatment needed between pdVWF and Veyvondi. Overall, yearly use was also similar, as were the length of hospital stays, and the median VWF dose per day of hospitalization.
When the researchers combined results from both Hopscotch-Will studies, they found that treatment patterns stayed stable over time, showing consistent use over the years.
The team assessed costs from the perspective of the French health insurance system for adults with VWD without inhibitors, or neutralizing antibodies that reduce the effectiveness of VWF replacement therapies. These results showed that Veyvondi costs significantly less per year and per patient than pdVWF treatment (€2,414 vs. €1,834 [about $2,800 vs. about $2,150).
We confirmed a trend toward lower consumption of [lab-made VWF replacement therapy] … and coupled with significantly lower overall costs.
In a separate analysis of seven people with VWD type 3, the most severe form of the disease, six patients (without inhibitors) received pdVWF during 10 hospital stays. In comparison, three patients received Veyvondi during four stays. Half of these hospitalizations were for noninvasive procedures, 29% for minor procedures, and 21% for major procedures, mainly for digestive bleeding or joint bleeding.
In this group, the median yearly treatment dose was higher with pdVWF than with Veyvondi (48 vs. 11 IU/kg per patient), according to the researchers.
The data to date indicate the two versions of VWF replacement therapy are equally effective, the scientists concluded. Further, “we confirmed a trend toward lower consumption of rVWF … and coupled with significantly lower overall costs,” the team wrote.
Still, the scientists noted that “further in-depth analyses at a larger scale will be required to confirm and refine these results.”
