FDA approves prophylaxis with Vonvendi in Type 3 von Willebrand disease.

Vonvendi is the only recombinant von Willebrand factor (VWF) replacement therapy, and the first and only treatment to reduce the frequency of bleeding episodes for severe Type 3 VWD approved by the FDA for routine prophylactic use. Vonvendi is now indicated for routine prophylaxis in adults with severe Type 3 VWD receiving on-demand therapy, as well as on-demand and perioperative bleed management in adults with VWD.

The approval is based on data from a prospective, open-label, international multicenter study to evaluate efficacy and safety of prophylactic treatment in reducing the frequency of bleeding episodes in 10 adult patients diagnosed with severe Type 3 VWD who were previously treated on-demand. Based on descriptive statistics, the median annualized bleeding rates (ABR) for all bleeds (treated and untreated spontaneous and traumatic bleeding events) was reduced from historical median ABR 5.0 (range: 3.0, 159.0) to an on-study median ABR of 2.3 (range: 0, 157.9), which is a 54.7% reduction. The most common adverse reactions (at least 2% of subjects) observed in adult patients treated with Vonvendi in clinical trials were headache, vomiting, nausea, dizziness, arthralgia, joint injury, vertigo, ALT increased and generalized pruritus.