The US Food and Drug Administration (FDA) has awarded breakthrough therapy designation to Roche’s Hemlibra, fast-tracking its development and review for the treatment of haemophilia A without factor VIII inhibitors.
Hemlibra is a prophylactic, subcutaneous, once-a-week drug formulated to combine the factor IXa and factor X to trigger coagulation cascade and restore blood clotting in haemophilia A.
The FDA previously granted similar status to Hemlibra in September 2015 for routine prophylaxis of bleeding episodes in adults and children with haemophilia A characterised by factor VIII inhibitors.
Hemlibra received complete regulatory approval in November last year.
Roche chief medical officer and global product development head Sandra Horning said: “Hemlibra is the first medicine to show superior efficacy compared to factor VIII prophylaxis, the standard of care for people with haemophilia A without inhibitors, in an intra-patient comparison.
“We look forward to working with health authorities to make Hemlibra available to people without inhibitors as soon as possible, and we are excited to share this news with the community as we join in celebrating World Hemophilia Day.”
The latest FDA decision comes after a review of findings from the Phase III HAVEN 3 trial where the drug demonstrated a statistically significant and clinically meaningful decrease in treated bleeds in subjects aged 12 or above when compared to no prophylaxis.
It was observed that once-weekly Hemlibra prophylaxis was superior to previous factor VIII.
Hemlibra also secured approvals in other international markets for routine prophylaxis of bleeding episodes in people with haemophilia A with factor VIII inhibitors.