29 May, 2018 New Clinical Results from APOLLO Phase 3 Study of Patisiran at ESC-HF
We presented new results from the APOLLO Phase 3 study of patisiran, an investigational RNAi therapeutic for the treatment of hereditary ATTR (hATTR) amyloidosis, at the European Society of Cardiology Heart Failure 2018 Congress, held May 26-29, 2018 in Vienna, Austria.
Merlini et al. – “Impact of Patisiran on Norfolk Quality of Life Questionairre Diabetic Neuropathy in Patients with Hereditary Transthyretin-Mediated Amyloidosis: Results from the Cardiac Subpopulation in the Phase 3 APOLLO Study”
NT-proBNP as a Biomarker
Based on the data from the APOLLO study, baseline NT-proBNP serum levels in hATTR amyloidosis patients are predictive of survival. These data underscore the importance of diagnosing and potentially treating patients early in the course of the disease.
Quality of Life in the Cardiac Subpopulation
These results show that treatment with patisiran led to improved quality of life compared to placebo in hATTR amyloidosis patients with evidence of cardiac involvement. These results are consistent with the outcomes in the overall study population, thereby demonstrating that patisiran potentially provides clinical benefit to hATTR amyloidosis patients with both polyneuropathy and cardiomyopathy.