Haemophilia is a rare genetic bleeding disorder that causes the blood to take a long time to clot because of a deficiency in one or several blood clotting factors. It is almost exclusively found in males. According to the World Federation of Hemophilia, more than 38,000 people worldwide were living with haemophilia B in 2021.
Pfizer’s BENEGENE-2 study met its primary endpoint of non-inferiority and superiority in the annualised bleeding rate (ABR) of total bleeds after a fidanacogene elaparvovec infusion versus prophylaxis regimen with Factor IX (FIX), administered as part of usual care.
