First up is Alnylam-partnered RNAi candidate fitusiran, an antithrombin-targeting agent in development as a treatment for haemophilia A and B with as few as six injections per year, making it a potential rival to Roche’s big-selling therapy Hemlibra (emicizumab).
New data from the ATLAS-PPX trial reported at ISTJ showed that fitusiran given as a once-monthly subcutaneous injection resulted in a 61% reduction in the annualised bleed rate (ABR) over seven-month follow-up period compared to prior therapy with either blood clotting factor replacement or bypassing agents (BPAs).
