Clinical Data
New analyses examined 21 total bleed events in 41 patients treated with fitusiran, after achieving greater than 75 percent antithrombin lowering. This analysis found that dosing of agents was generally within the normal dose range for replacement Factor VIII and Factor IX as well as for the bypassing agent rFVIIa, while at the lower end of the range for the bypassing agent aPCC. Treatment of all breakthrough bleed events resulted in successful hemostasis without any thromboembolic events.


An evaluation of fitusiran at refrigerated (i.e., 5ºC), customary room temperature (i.e., 25ºC/60 percent relative humidity) and at accelerated aging conditions (i.e., 40ºC) demonstrated a robust stability profile, with all key quality attributes predicted to be retained at both refrigerated and room temperature conditions for at least 24 months. Further, fitusiran has shown resistance to thermal stress and cyclic temperature fluctuations that may occur during real world storage and handling of the drug product. Consequently, the stability profile of fitusiran could enable convenient transportation and storage, including in parts of the world where cold chain delivery is a challenge.