Ready to contact us for a consultation about your condition and our medical services? The staff of our Global Patient Services is ready to help. Please select the appropriate button to get started.

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

Hemophilia research

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.”

Sheba DUNS 100

For The Second Consecutive Year: Sheba Tops the Healthcare Sector on Dun & Bradstreet’s List of Leading Organizations

Dun & Bradstreet, the world’s foremost commercial data company, ranked 71 doctors from Sheba Medical Center in its list of the top Israeli clinical experts.…
Read More

Ayelet’s Miracle: The First Woman to Conceive Four Children Following Ovarian Tissue Cryopreservation

At 21, Ayelet Rozenberg was diagnosed with Hodgkin’s lymphoma. Facing aggressive therapies necessary to save her life, Ayelet feared she would never be able to…
Read More
bowel disease
News Sep 07.

Sheba Partners with Biomica to Advance Inflammatory Bowel Disease Care

As part of the new collaboration, Sheba and Biomica will engage in joint microbiome clinical research to develop better treatments for inflammatory bowel disease (IBD)…
Read More