VEYVONDI (vonicog alfa) - Von Willebrand disease (VWD)

The clinical benefit of VEYVONDI (vonicog alfa) is substantial in long-term prophylactic therapy in adults (age 18 years and older) with von Willebrand disease, when desmopressin (DDAVP) treatment alone is ineffective or contraindicated.

Considering:

• clinical data from the pivotal phase 3 study 071301 suggesting the efficacy of long-term prophylactic therapy using VEYVONDI (vonicog alfa) in adults with a severe form of von Willebrand disease, with low mean treated annualised bleeding rates under treatment,
• the non-optimal quality of this evidence, which is a source of uncertainties with respect to the effect observed, in view of the methodological weaknesses of the study, in particular the open-label and non-randomised design, the descriptive character of the results, the low patient numbers assessed, and the numerous premature withdrawals and major protocol deviations reported,
• the absence of robust comparisons with the available therapeutic alternatives,
• the safety profile deemed to be acceptable and consistent with that established in the other indications that are already reimbursable,

the Committee deems that VEYVONDI (vonicog alfa) powder and solvent for solution for injection provides no clinical added value (CAV V) in the current care pathway for long-term prophylactic therapy, which includes relevant comparators (medicinal products containing plasma-derived VWF alone or combined with factor VIII). Furthermore, the Committee
maintains the conclusions indicated in its opinion of 12 December 2018 in the other MA indications (no clinical added value (CAV V) in the care pathway).