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New Sheba Study Indicates: Novel Treatment Sharply Reduces Bleed Rates Among Hemophilia Patients

Hemophilia research on Fitusiran RNAi therapy

While receiving the Fitusiran RNAi therapy, more than 63% of study participants experienced no bleeds for over seven months.

Developed by Sanofi Genzyme and Alnylam Pharmaceuticals, the Fitusiran RNAi therapy was designed to promote blood clotting by lowering the levels of a protein called antithrombin, which normally prevents clotting.

According to a study conducted at Sheba to evaluate the efficacy and safety of the Fitusiran therapy for hemophilia A and B patients, 63.1% of the 80 participants experienced no bleeding for over seven months while receiving the treatment on a monthly basis.

Dr. Dietmar Berger, Head of Global Development and Chief Medical Officer at Sanofi: “These findings support Fitusiran’s potential to transform treatment for people with hemophilia A or B, with or without inhibitors.”

The open-label phase 3 trial included 80 male participants with either hemophilia type A or B who were receiving approved prophylactic (preventive) therapies. Over the first six months of the trial, participants continued their standard prophylactic therapy regimen, which included 2-3 infusions of clotting factors each week. Then, for a total of seven months, the patients switched to a once-a-month Fitusiran 80 mg regime, administered via subcutaneous injections.

Among participants who received Fitusiran, the median annual bleeding rate was zero, while only less than 1 in 5 (16.9%) of those who received the prophylactic therapy remained bleed-free.

According to Head Trial Investigator, Prof. Gili Kenet, who also serves as Director of the Israeli National Hemophilia Center at Sheba: “These phase 3 results are encouraging and support Fitusiran’s potential to provide people with hemophilia A or B, regardless of inhibitor status, with a meaningful reduction in bleeding episodes.”

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