Irish biotechnology firm Shire has secured authorisation from the European Commission (EC) to market Veyvondi for the treatment of adults with von Willebrand disease (VWD).
The drug is indicated for bleeding events and the treatment or prevention of surgical bleeding in patients who do not respond to or cannot use desmopressin (DDAVP) therapy alone.
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VWD is a common, genetic bleeding disorder caused by a deficiency or dysfunction of von Willebrand Factor (VWF), which is required for normal blood clotting.
Veyvondi is a recombinant von Willebrand Factor (rVWF) therapy. It is designed to selectively target the primary deficiency or dysfunction of VWF.
The therapeutic also enables the body to restore and maintain sufficient levels of Factor VIII (FVIII) in plasma.
Shire chief scientific officer and Research and Development head Andreas Busch said: “The approval in Europe for Veyvondi marks a key milestone in our efforts to tackle unmet medical needs for those living with von Willebrand disease.
“We are excited to take the next steps in ensuring that Veyvondi is widely available across Europe to address the individual needs of those affected by the condition and in need of factor replacement.”
The EC decision comes after review of results from a Phase I and two Phase III clinical trials conducted in a total of 80 subjects.
During the multi-centre, controlled, randomised, single-blind, dose-escalation Phase I trial, the safety, tolerability and pharmacokinectics (PK) of rVWF:rFVIII were studied in severe VWD patients aged between 18 and 60 years old.
Meanwhile, a multi-centre open-label Phase III study evaluated the PK, safety and efficacy of rVWF:rFVIII and rVWF while treating bleeding episodes in adult subjects.
A separate prospective, open-label, uncontrolled, non-randomised, international multi-centre Phase III trial investigated the haemostatic efficacy and safety of rVWF with or without rFVIII in adult patients undergoing major, minor or oral elective surgical procedures.
