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Investigational once-weekly BIVV001 (efanesoctocog alfa) prophylaxis met the primary efficacy endpoint providing clinically meaningful bleed protection for people with severe hemophilia A.
SAR-439774 (fitusiran) is an investigational, subcutaneously administered small interference RNA therapeutic in development for the prophylactic treatment of people with hemophilia A or B, with or without inhibitors.
BIVV001 (efanesoctocog alfa) is a novel and investigational factor VIII therapy designed to provide normal to near-normal factor activity levels for the majority of the week in a once-weekly prophylactic treatment regimen.
The study met the primary endpoint, showing a clinically meaningful prevention of bleeds in people with severe hemophilia A receiving weekly prophylaxis with BIVV001 (efanesoctocog alfa) over a period of 52 weeks.
Data from phase 3 studies achieved primary and secondary endpoints for ALN-AT3SC (Fitusiran) prophylaxis demonstrated significant and clinically meaningful improvements in bleed protection across all study populations.
Efanesoctocog alfa, previously known as BIVV001, is an investigational factor VIII replacement therapy that has the potential to transform therapy and provide high sustained factor activity levels for people with hemophilia A.
Sanofi will resume fitusiran dosing in ongoing U.S. adolescent and adult clinical studies. Fitusiran is an investigational, small interference RNA therapy in development for the treatment of people with hemophilia A or B, with or without inhibitors.
In patients with late-onset Pompe disease (LOPD), 5.5 years of safety and exploratory efficacy results showed avalglucosidase alfa was well-tolerated and stabilized pulmonary and motor function.
The WFH, EHC and NHF have learned of and subsequently confirmed a decision by Sanofi Genzyme to initiate a voluntary sponsor-led global dosing hold on its full clinical development program for fitusiran due to the identification of new adverse events.
The MAA is based on positive data from two trials: Phase 3, pivotal COMET trial, which evaluated the safety and efficacy of avalglucosidase alfa compared to alglucosidase alfa (standard of care) in patients with late-onset Pompe disease, and Phase 2 mini-COMET trial.
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