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Alnylam Announces New Data from ATTR Amyloidosis Programs at the Peripheral Nerve Society’s 2021 Annual Meeting
− Phase 3b Open-Label Study Showed Treatment with Patisiran Achieved Rapid and Sustained Reduction in Serum TTR Levels in hATTR Amyloidosis Patients with
About this update from Alnylam Pharmaceuticals, Inc.
[{"type":"text","content":"\n− Phase 3b Open-Label Study Showed Treatment with Patisiran Achieved Rapid and Sustained Reduction in Serum TTR Levels in hATTR Amyloidosis Patients with Polyneuropathy Progression Following Orthotopic Liver Transplant – \n\n− Additional Results from Pre-specified Patient Subgroups Analysis Included with Encore Presentation of HELIOS-A Phase 3 Study of Investigational Vutrisiran −\n\n CAMBRIDGE, Mass.--(BUSINESS WIRE)--\nAlnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today announced positive results from a Phase 3b open-label study conducted to evaluate the safety, efficacy and pharmacokinetics (PK) of patisiran in hereditary ATTR (hATTR) amyloidosis patients with polyneuropathy progression after receiving an orthotopic liver transplant (OLT). 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. In addition, the safety profile of patisiran was consistent with the previously reported safety results observed in the APOLLO Phase 3 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.\n\nIn addition, positive results from the HELIOS-A Phase 3 study of vutrisiran, an investigational RNAi therapeutic in development for the treatment of transthyretin-mediated (ATTR) amyloidosis, were presented today with additional data from pre-specified patient subgroups. Improvement in the modified Neuropathy Impairment Score (mNIS+7) and Norfolk Quality of Life-Diabetic Neuropathy score (Norfolk QOL-DN) from vutrisiran treatment was consistently observed across all pre-specified patient subgroups, including age, sex, race, geographic region, baseline neuropathy impairment, genotype, prior TTR stabilizer use, baseline Familial Amyloid Polyneuropathy (FAP) stage, and in a pre-specified cardiac subpopulation. These data were presented as posters during the 2021 ...