Sanofi receives US approval for hemophilia A and B therapy
The US Food and Drug Administration (FDA) has approved Sanofi's Qfitlia (fitusiran), the first antithrombin-lowering therapy for hemophilia, offering a new treatment option with infrequent dosing.
Sanofi announced that the US Food and Drug Administration (FDA) has approved Qfitlia (fitusiran), a novel therapy for hemophilia A or B. This marks the first approval for an antithrombin-lowering treatment aimed at routine prophylaxis to prevent or reduce bleeding episodes in adult and pediatric patients aged 12 and older, with or without inhibitors.
The approval, based on data from the ATLAS phase 3 studies, demonstrated clinically meaningful bleed protection as measured by annualized bleeding rates (ABR). Qfitlia's unique mechanism works by lowering antithrombin, a protein that inhibits blood clotting. This action helps to increase thrombin generation, thereby restoring hemostasis in individuals with hemophilia.
Utilizing small-interfering RNA technology, Qfitlia allows for a low treatment frequency, administered via subcutaneous injection with a prefilled pen or vial and syringe. Patients can receive the therapy as few as six times per year. This infrequent dosing schedule aims to reduce the treatment burden for patients and their families compared to existing treatment options.
Sanofi stated that Qfitlia has the potential to significantly alter the hemophilia treatment landscape. The company highlighted that the therapy offers effective bleed protection, infrequent dosing, and simplified administration. Qfitlia is positioned to provide the fewest doses among all prophylactic therapies, with a price point comparable to other hemophilia prophylactic treatments. The FDA had previously granted Qfitlia Orphan Drug Designation and Fast Track Designation.