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P.024 Long-term use of patisiran in patients with hereditary transthyretin amyloidosis (hATTR): 12 month efficacy & safety data from a global open label extension (OLE) study

  • A Gonzalez-Duarte (a1), T Coelho (a2), D Adams (a3), C Yang (a4), M Polydefkis (a5), A Kristen (a6), I Tournev (a7), H Schmidt (a8), J Berk (a9), K Lin (a4), PJ Gandhi (a9), M Sweetser (a9), M White (a9), J Gollob (a9), OB Suhr (a10) and APOLLO Study Investigators (a1) (a2) (a3) (a4) (a5) (a6) (a7) (a8) (a9) (a10)...

Abstract

Background: Hereditary transthyretin-mediated (hATTR) amyloidosis is a multi-systemic, heterogenous, life-threatening disease. Patisiran resulted in significant improvement in neuropathy and QoL at 18-months compared to placebo, and was generally well-tolerated in the Phase 3 APOLLO study. Methods: Multi-center, OLE study to evaluate the efficacy and safety of long-term patisiran dosing for ≤ 5 years in hATTR amyloidosis patients with polyneuropathy who have completed the APOLLO study (NCT02510261). Endpoints include safety, tolerability and long-term efficacy of patisiran. Measures of clinical benefit are the same endpoints used in APOLLO including changes in mNIS+7 composite neuropathy impairment score and QoL (Norfolk QoL-DN) Results: As of December 2017, 184 of 186 (99%) patients who completed APOLLO and 25 patients from the Ph 2 OLE study enrolled in the Global OLE study. Baseline data for 211(APOLLO/placebo, n=49; APOLLO/patisiran, n=137 and patisiran Ph 2 OLE, n=25) patients included: median age 61 years (26-84); 74% males; 46% V30M. Interim safety data and 12-month efficacy results will be presented. Conclusions: The global OLE study includes a diverse population of hATTR amyloidosis patients. Interim data will include the long-term safety and maintenance of effect in patients continuing on patisiran, as well as the impact of treatment with patisiran on patients previously treated with placebo.

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P.024 Long-term use of patisiran in patients with hereditary transthyretin amyloidosis (hATTR): 12 month efficacy & safety data from a global open label extension (OLE) study

  • A Gonzalez-Duarte (a1), T Coelho (a2), D Adams (a3), C Yang (a4), M Polydefkis (a5), A Kristen (a6), I Tournev (a7), H Schmidt (a8), J Berk (a9), K Lin (a4), PJ Gandhi (a9), M Sweetser (a9), M White (a9), J Gollob (a9), OB Suhr (a10) and APOLLO Study Investigators (a1) (a2) (a3) (a4) (a5) (a6) (a7) (a8) (a9) (a10)...

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