Shire gets EC marketing authorisation for 'VEYVONDI' treatment

Rare diseases specialist Shire announced on Wednesday that the European Commission has granted marketing authorisation for ‘VEYVONDI’, for the treatment of bleeding events and the treatment and prevention of surgical bleeding in adults with von Willebrand disease, when desmopressin treatment alone is ineffective or not indicated.

The FTSE 100 biotech firm said VEYVONDI was the “first and only” recombinant von Willebrand Factor treatment in the EU for von Willebrand disease that specifically addressed the primary deficiency or dysfunction of von Willebrand Factor, while also allowing the body to restore and maintain adequate Factor VIII plasma levels.

“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,” said Shire’s head of research and development and chief scientific officer Andreas Busch.

“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.”

Shire said the authorisation was based on outcomes from three clinical trials of a total 80 patients with von Willebrand disease exposed to VEYVONDI.

They included a Phase 1 multicentre, controlled, randomised, single-blind, dose-escalation study of the safety, tolerability and pharmacokinetics of rVWF:rFVIII in subjects aged 18 to 60 years with severe von Willebrand disease; a Phase 3 multicentre, open-label study to assess the pharmacokinetics, safety and efficacy of rVWF:rFVIII and rVWF in the treatment of bleeding episodes in adult subjects with severe von Willebrand disease; and a Phase 3, prospective, open-label, uncontrolled, non-randomised, international multicentre study to assess the hemostatic efficacy and safety of rVWF with or without rFVIII in 15 adult subjects with severe von Willebrand disease undergoing major, minor, or oral elective surgical procedures.

It said von Willebrand disease was the most common inherited bleeding disorder, affecting up to 1% of the global population, or approximately 100,000 people in the EU.

It was caused by a deficiency or dysfunction of von Willebrand Factor - one of several types of proteins in the blood that were needed to facilitate proper blood clotting.

Only a minor proportion of affected individuals had the severe form of the disease and were in need of VWF replacement, the company explained.

Symptoms ranged from nosebleeds to bleeding from the gums and easy bruising.

Bleeding from the stomach and intestines could also occur, but were less common.

With the approval, Shire said it was now authorized to market VEYVONDI in the 28 current member states of the European Union, as well as in Iceland, Liechtenstein and Norway.