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Two fitusiranPhase 3 studies published in The Lancet and The LancetHaematologyhighlight potential to address unmet needs across all types of hemophilia
Paris–April 4, 2023– Two studies, published in The Lancetand The LancetHaematology, evaluating the efficacy and safety of fitusiran, an investigational siRNA therapy for the prophylactic treatment of adults and adolescents with hemophilia A or B, reinforce the potential of this investigational therapy to transform the current standard of care and address unmet needs for all types of hemophilia, regardless of inhibitor status.
Hemophilia A and B are rare congenital lifelong bleeding disorders in which the ability of a person’s blood to clot is impaired, leading to excessive bleeds and spontaneous bleeds into joints that can result in joint damage and chronic pain, and significantly impact quality of life. Fitusiran has the potential to provide prophylaxis for all types of hemophilia, regardless of inhibitor status, with as few as six subcutaneous injections per year.
Dietmar Berger, M.D., Ph.D.Head of Global R&D ad interim and Chief Medical Officer at Sanofi“Sanofi is committed to advancing the standardof care for all people with hemophiliathrough innovative science,providingconsistent bleed protectionwhile reducing treatment burden.We are entering a new erainhemophilia where,for the first time, peoplecan choose therapies that meet their personal needs.These published data validateour science and add to a growing body of evidence supporting fitusiran’spotential totransform the treatmentlandscape. We look forward to sharing additional data on fitusiranlater this year.”
Both Phase 3 studies compared once-monthly subcutaneous fitusiran prophylaxis (80mg) with on-demand/episodic use of clotting factor concentrates in the ATLAS-A/B study, and on-demand/episodic use of bypassing agents in the ATLAS-INH study. Across both clinical studies, prophylactic treatment with fitusiran reduced annualized bleeding rates by 90% (95% CI [84.1%; 93.6%], P
