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Patisiran for hATTR amyloidosis: 24-month efficacy and safety

AAN 2021
In patients with hereditary transthyretin-mediated (hATTR) amyloidosis with polyneuropathy, patisiran continued to demonstrate durability of efficacy after 24 months of treatment in the ongoing open-label extension (OLE) of the APOLLO study.

APOLLO (NCT01960348) is the largest randomised clinical trial of patients with hATTR amyloidosis with polyneuropathy to date. Patients were eligible to enter the OLE if they had completed the parent study, notably APOLLO participants randomised to placebo (APOLLO/placebo, n=49) or patisiran (APOLLO/patisiran, n=137), as well as phase 2 OLE patients (n=25) [1]. For 178 patients, 24-month data was available.

Modified Neuropathy Impairment Score +7 (mNIS+7) demonstrated durable improvement. Mean change in the APOLLO/patisiran and phase 2 OLE groups was -5.9 and -4.9, respectively, compared with baseline scores in the parent study. Norfolk Quality of Life-Diabetic Neuropathy (QOL-DN) also continued to show durable improvement in APOLLO/patisiran patients (mean change -2.4). The safety profile of patisiran remained consistent with previous studies.

Another presentation described the impact of patisiran on functioning in daily life of 225 APOLLO participants [2]. For the majority of these, patisiran preserved the ability to perform activities of daily living and functional status versus placebo. The odds of stabilising or improving these assessments were also higher.

  1. Adams D, et al. Global Open-label Extension: 24-month Data in Patients with hATTR Amyloidosis. S32.001, AAN 2021 Virtual Congress, 17-22 April.
  2. Peltier A, et al. Impact of Patisiran on Activities of Daily Living and Functional Status in hATTR Amyloidosis. S32.002, AAN 2021 Virtual Congress, 17-22 April.

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