10 Dec, 2017 Fitusiran and Givosiran Updates at ASH Annual Meeting
We presented new data for fitusiran and givosiran at the American Society of Hematology 59th Annual Meeting & Exposition in Atlanta.
Sardh et al. presented updates from EXPLORE, the first international, natural history study in patients with acute hepatic porphyrias (AHPs). Results demonstrate that patients suffer from acute and severe disabling attacks, 75% of which required treatment at a healthcare facility or with intravenous hemin. Moreover, 65% of patients also reported chronic symptoms (most commonly pain), both during and in between attacks, which collectively result in a highly diminished quality of life. Given the continued morbidity and mortality there remains a high unmet need for an efficacious, simple, fast-acting and better tolerated treatment for patients with AHPs.
Negrier et al. described perioperative hemostatic management during dental/surgical procedures in patients with hemophilia receiving fitusiran, an investigational RNAi therapeutic, in clinical trials. Successful perioperative hemostatic management with factor or bypassing agent (BPA) in the context of antithrombin (AT) lowering with fitusiran was observed. Further, these cases suggest that the hemostatic effect of fitusiran may allow for reduced dosing of factor or BPA for perioperative management.
Sridharan et al. demonstrated the use of a computational model to describe the clinically observed relationship between AT lowering and thrombin generation. The model may be used to support the use of lower doses of factor to treat breakthrough bleeds while being treated with an AT lowering agent. Additionally, based on model predictions, AT lowering may potentially be a therapeutic strategy for certain rare bleeding disorders, including factor V, VII, or X deficiencies.