Shire plc,global biotech leader in rare diseases has announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has issued a positive opinion recommending the granting of marketing authorization in the European Union (EU) for VEYVONDI, for the treatment of bleeding events and treatment/prevention of surgical bleeding in adults (age 18 and older) with von Willebrand disease (VWD) when desmopressin (DDAVP) treatment alone is ineffective or not indicated.
VEYVONDI should not be used in the treatment of Hemophilia A. If approved, VEYVONDI will be the first and only recombinant von Willebrand Factor (rVWF) treatment in the EU for von Willebrand disease (VWD) that specifically addresses the primary deficiency or dysfunction of von Willebrand Factor (VWF) while also allowing the body to restore and maintain adequate Factor VIII (FVIII) plasma levels.
“This positive opinion moves us one step closer to bringing the first, targeted recombinant therapy to people living with von Willebrand disease who still have unmet medical needs,” said Andreas Busch, Head of Research and Development and Chief Scientific Officer, Shire. “As we continue our long-standing commitment to address unmet needs for people with bleeding disorders, we welcome the potential for individualized treatment of patients with VWD in need of replacement therapy.”
The CHMP submission was based on outcomes from three clinical trials of a total 80 patients with VWD exposed to VEYVONDI. These include a Phase 1 multicenter, controlled, randomized, single-blind, dose-escalation study of the safety, tolerability and pharmacokinectics (PK) of rVWF:rFVIII in subjects 18 to 60 years of age with severe VWD; a Phase 3 multicenter, open-label study to assess the PK, safety and efficacy of rVWF:rFVIII and rVWF in the treatment of bleeding episodes in adult subjects with severe VWD; and a Phase 3, prospective, open-label, uncontrolled, non-randomized, international multicenter study to assess the hemostatic efficacy and safety of rVWF with or without rFVIII in 15 adult subjects with severe VWD undergoing major, minor, or oral elective surgical procedures.
VWD is the most common inherited bleeding disorder, affecting up to 1 percent of the global population or approximately 100,000 people in the EU. VWD is caused by a deficiency or dysfunction of VWF, one of several types of proteins in the blood that are needed to facilitate proper blood clotting. Only a minor proportion of affected individuals have the severe form of the disease and are in need of VWF replacement.
The CHMP's positive opinion will be reviewed by the European Commission, which has the authority to grant marketing authorization in the EU.