Two fitusiran phase III studies published in The Lancet and The Lancet Haematology highlight potential to address unmet needs across all types of hemophilia
Two studies, published in The Lancet and The Lancet Haematology, 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.
Both Phase III 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 <0.0001) compared to the control arms, showing a statistically significant and clinically meaningful improvement in bleeding episodes when compared to on-demand treatments; and showed improvement in quality of life.
In the study ATLAS-INH study published by The Lancet, 66% of participants with inhibitors (25 out of 38) receiving fitusiran 80mg monthly experienced zero bleeding episodes compared to 5% (1 out of 19) receiving an on-demand bypassing agent after nine months of treatment.
The ATLAS A/B study published in The Lancet Haematology showed 51% of participants without inhibitors (40 out of 79) who received fitusiran 80mg monthly prophylaxis experienced zero bleeds compared to 5% (2 out of 40) in the comparator group, receiving on-demand clotting factor concentrates.
Sanofi is currently investigating the efficacy and safety of fitusiran under a revised regimen which includes lower doses and less frequent dosing (as few as six subcutaneous injections per year), maintaining an antithrombin target range of 15-35% in all ongoing studies.
See-"Efficacy and safety of fitusiran prophylaxis in people with haemophilia A or haemophilia B with inhibitors (ATLAS-INH): a multicentre, open-label, randomised phase III trial";Young, Guy et al.-Published:March 29, 2023DOI:https://doi.org/10.1016/S0140-6736(23)00284-2.
See- "Fitusiran prophylaxis in people with severe haemophilia A or haemophilia B without inhibitors (ATLAS-A/B): a multicentre, open-label, randomised, phase III trial" The Lancet Haematology Published: March 29, 2023; Alok Srivastava and others.-Published:March 29, 2023DOI:https://doi.org/10.1016/S2352-3026(23)00037-6.
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