Roche has received approval from the European Commission (EC) for the use of Hemlibra (emicizumab) in routine prophylaxis of bleeding episodes in severe haemophilia A patients without factor VIII inhibitors.
The drug is a bispecific antibody targeting factor IXa- and factor X. It combines the factor proteins to stimulate coagulation cascade and restore the blood clotting process.
Hemlibra was created by Chugai Pharmaceutical, which is co-developing the drug in alliance with Roche and Genentech.
The EC approval comes after review of data from the Phase III HAVEN 3 and HAVEN 4 clinical trials performed in adults and adolescents aged 12 years or older.
In HAVEN 3, prophylaxis with the drug demonstrated statistically significant and clinically meaningful decrease in treated bleeds, compared to no prophylaxis as well as to previous therapy with factor VIII prophylaxis.
Roche noted that Hemlibra is the first to significantly mitigate treated bleeds compared to prior factor VIII prophylaxis, the standard of care for haemophilia A without factor VIII inhibitors.
During the HAVEN 4 trial, the drug led to a clinically meaningful control of bleeding in participants who received dosing every four weeks.
Roche chief medical officer and Global Product Development head Sandra Horning said: “Today’s approval is a landmark moment as Hemlibra is the first new class of treatment for people with severe haemophilia A without inhibitors in nearly 20 years.
“We will continue to work with EU member states, to bring this important treatment to those in need as quickly as possible.”
The drug obtained the US Food and Drug Administration (FDA) approval in October last year for use in routine prophylaxis to prevent or reduce the frequency of bleeding episodes in haemophilia A patients without factor VIII inhibitors.
The drug holds approval in more than 60 countries for routine prophylaxis in in people with haemophilia A with factor VIII inhibitors.