The P-III (ATLAS-PPX) study evaluated fitusiran (80 mg, qm) in 80 adults & adolescents aged ≥12yrs. with sev. hemophilia A or B with/out inhibitors who were prior treated with a prior factor or BPA prophylaxis
The study met its 1EPs and showed a 61% reduction in bleeding episodes over 16.9% with prior factor or BPA prophylaxis; patients experienced zero treated bleeds (63.1% vs 16.9%); m-ABR for treated bleeds was 0.0 with/out inhibitors vs 6.5 and 4.4 for patients with/out inhibitors, respectively on prior prophylaxis.
67 patients were exposed to one dose of fitusiran. The results were consistent with the prior identified risk of fitusiran, 2 patients (3.0%) reported suspected or confirmed thromboembolic events
Ref: GlobalNewswire | Image: Sanofi
