The P-III (APOLLO-B) trial evaluate patisiran (0.3mg/kg) in a ratio (1:1) in 360 patients with ATTR amyloidosis (hereditary or wild-type) with cardiomyopathy at 69 sites in 21 countries
The trial met its 1EPs & 2EPs i.e., change from baseline in 6-MWT @12mos. & health-related QoL & also showed a composite of all-cause mortality, frequency of CV events & change from baseline in 6-MWT; frequency of all-cause hospitalizations & urgent HF visits in patients not on tafamidis at baseline & overall study population along with encouraging safety & tolerability profile
The exploratory endpoints incl. cardiac biomarkers & multiple imaging tools to characterize the burden of cardiac involvement. The company plan to submit the sNDA of patisiran with the US FDA in late 2022
Ref: Alnylam | Image: Alnylam
