Phase IIIb open-label study of Onpattro shows benefit in hereditary ATTR amyloidosis.- Alnylam Pharmaceuticals

In patients treated with patisiran, the median reduction in serum TTR levels compared to baseline was 91 percent, measured as an average of the month six and month 12 reduction. Results from the study show that at month 12, patisiran treatment resulted in an improvement in neuropathy, as demonstrated by a 3.7 point decrease in the mean total neurological impairment (NIS) score from baseline. Patisiran treatment also resulted in an improvement in quality of life with a 6.5 decrease in mean total Norfolk Quality of Life-Diabetic Neuropathy score (Norfolk QOL-DN) and autonomic symptoms with treatment resulting in a 5.0 decrease in the least squares (LS) mean total COMPASS-31 score from baseline. Patisiran treatment also demonstrated stabilization in other endpoints, including measures of disability like the Rasch-built Overall Disability Scale (R-ODS) and nutritional status with the modified body mass index (mBMI) which were both stable relative to baseline at month 12. Patisiran also demonstrated an encouraging safety and tolerability profile after 12 months of treatment and there were no drug-related study discontinuations or deaths. In addition, the safety profile of patisiran was consistent with the previously reported safety results observed in the APOLLO Phase III study. Patisiran is the established name for Onpattro, which is approved in the United States, Canada and Japan for the treatment of the polyneuropathy of hATTR amyloidosis in adults, and in the European Union, Switzerland and Brazil for the treatment of hATTR amyloidosis in adults with Stage 1 or Stage 2 polyneuropathy. These data will form the basis of post-approval supplements which have the potential to change labeling for Onpattro where approved, including in the European Union.