Sanofi presents at EAHAD amended protocols for fitusiran clinical studies for haemophilia A + B.

Phase III studies of fitusiran were put on hold late 2020 because of side effect concerns including non-fatal thrombotic events, but the studies have now resumed, Sanofi highlighted in its 2020 financial results reporting on 5 February. The amended protocol being implemented for all ongoing adult and adolescent fitusiran clinical studies was presented at the 14th Annual Congress of the European Association for Haemophilia and Allied Disorders (EAHAD). Fitusiran is an investigational, subcutaneously administered small interference RNA therapy in development for the treatment of people with hemophilia A or B, with or without inhibitors. Implementation of the amended protocol follows Sanofi’s voluntary pause in dosing and enrollment in the ongoing fitusiran clinical studies on October 30, 2020 to allow the investigation of reports of non-fatal thrombotic events in the trials. This assessment included analysis of reported thrombotic events, anti-thrombin levels, and other available clinical data. Sanofi’s priority is the safety of patients. Amendments to the dose and dosing regimen, aimed at further enhancing the benefit-risk profile of fitusiran for patients, were highlighted in the oral presentation. Under the amended protocol, the dose for adults and adolescents will be reduced to 50 mg every other month (six times a year), with the potential to adjust the dose and/or dose frequency based on an individual patient’s anti-thrombin levels.