Alnylam's Fitusiran Could Provide Comprehensive Hemophilia Therapy Option
Fitusiran's mechanism of reducing antithrombin levels could apply to all hemophilia patients, as well as those with other rare bleeding disorders. Reducing antithrombin increases thrombin levels and helps to increase normal clotting.
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The RNAi pioneer voluntarily suspended dosing of the hemophilia candidate to consider trial protocol alterations after a patient receiving fitusiran in an open-label, Phase II trial died following a thrombosis event.
Updates for a range of new therapies from Roche, Alnylam, BioMarin and Spark Therapeutics at the ISTH meeting in Berlin show progress in development of new hemophilia alternatives, with more late-stage programs kicking off.
While attention has been focused on other products, Alnylam is set to take another RNAi product candidate – givosiran – into pivotal testing following positive early data in acute hepatic porphyria.