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Alnylam Presents New Long-Term Results from Global Open-Label Extension Study of ONPATTROAlnylam Presents New Long-Term Results from Global Open-Label Extension Study of ONPATTRO® (patisiran) at the European Academy of Neurology Virtual Congress 2020May 22, 2020 Patisiran Continues to Demonstrate Reversal of Neuropathy Progression, Improvement in Quality of Life, and Consistent Safety Profile with Additional 24 Months of Treatment in Global Open-Label Extension (OLE) Study Interim Data Presented on Patisiran Treatment in Patients with Disease Progression After an Orthotopic Liver Transplant “In collaboration with our clinical investigators, we are pleased to have the opportunity to share data from our patisiran clinical program in two abstracts at this year’s EAN virtual conference. In particular, results from our Global OLE study demonstrate sustained benefit for patients with hATTR amyloidosis with polyneuropathy treated with ONPATTRO, with maintained improvement in polyneuropathy symptoms and quality of life after an additional 24 months of treatment. As of Updated Results from Global OLE Study 24-month interim results were presented from the ongoing Global OLE study of patisiran evaluating the drug’s long-term efficacy and safety in eligible patients (N=211) who completed the Phase 2 OLE (N=25) and APOLLO Phase 3 (N=186) studies. The data shared include 178 patients who had 24 months or greater of exposure as of an For APOLLO placebo patients subsequently treated with patisiran for 24 months in the OLE study, neuropathy progression was also notably halted and QOL improved. However, these patients had experienced rapid progression while on placebo in the APOLLO study and did not return to their baseline scores, highlighting the importance of patients starting treatment with patisiran early. The long-term safety profile of patisiran was consistent with that observed and previously reported in the APOLLO Phase 3 study and the Phase 2 OLE study. ONPATTRO in Patients with Disease Progression Post-Orthotopic Liver Transplant In addition, data were presented from an interim analysis of the Phase 3b open-label study conducted across several European countries to evaluate the safety, efficacy and pharmacokinetics (PK) of patisiran in patients with hATTR amyloidosis with disease progression after receiving an orthotopic liver transplant (OLT). Historically, OLT has been used to slow disease progression in patients with early stages of hATTR amyloidosis; however, some patients experience disease progression after the transplant due to continued amyloid deposition of wild-type TTR on top of existing amyloid deposits in tissues. Twenty-three patients who showed disease progression post-OLT (based on an increase in polyneuropathy disability [PND] score) received patisiran infusion (0.3 mg/kg) every three weeks. After 3 weeks of patisiran treatment, the mean reduction from baseline in serum TTR levels was 81.9 percent. At the time of interim safety analysis (data cutoff as of To view the data presented at EAN, please visit www.alnylam.com/capella. |
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