European regulatory advisors have given their backing to Shire’s Adynovi (antihemophilic factor recombinant PEGylated), a haemophilia A treatment for adults and adolescents aged 12 and over.
The injectable is a new form of Advate – a haemophilia A treatment Shire acquired from Baxalta in a $32bn takeover – and offers twice-weekly prophylactic use as well as on-demand control of bleeding episodes.
Howard Mayer, senior vice president and ad-interim head, global research and development, said: “This positive opinion marks an important step in providing adults and adolescents with haemophilia A in Europe with the potential for a new treatment option with a twice-weekly prophylaxis dosing schedule.”
The Committee for Medicinal Products for Human Use (CHMP)’s go-ahead was based on positive outcomes of phase III clinical trials, which assessed the injectable in haemophilia A patients between ages 12 – 65.
Mayer added: “This means that patients can manage their haemophilia A care with their physicians in the way that works best for them.”
A designated orphan disease, haemophilia A is a rare bleeding disorder that causes longer-than-normal bleeding due to lack of clotting factor VIII in the blood.
The disorder affects more than 150,000 people across the globe and of the worldwide haemophilia patient population, it is estimated that 75% lack adequate treatment or access to treatment altogether.
The Irish biotech will now await full European approval for Adynovi, with the European Commission usually granting this within three months of a positive CHMP opinion.