Primary Endpoint: 1,2
- Change from baseline at 18 months vs placebo in modified Neuropathy Impairment Score + 7 (mNIS+7)
- mNIS+7 is an objective 304-point assessment of polyneuropathy in hereditary transthyretin-mediated (hATTR) amyloidosis
Change in mNIS+7 score1,a-c
- The progression of polyneuropathy in the placebo arm was consistent with the natural progression of the disease described in previous studies3-5
- Maintenance of efficacy was demonstrated for up to 36 months in 2 OLE studies6
aMean mNIS+7 at baseline was 80.9 with ONPATTRO and 74.6 with placebo.1 bBars represent SEM. cN=number of evaluable patients. d95% CI: -20.7, -11.3.1 e95% CI: -39.9, -28.1.1
CI=confidence interval; LS=least squares; OLE=open-label extension; SEM=standard error of the mean.
Reversal in neuropathy
from baseline at 18 months3,f,g
From New England Journal of Medicine, Adams D, Gonzalez-Duarte A, O’Riordan WD, et al,
"Patisiran, an RNAi Therapeutic, for Hereditary Transthyretin Amyloidosis," 379(1).
Copyright © 2018 Massachusetts Medical Society. Reprinted with permission from Massachusetts Medical Society.
fOdds ratio: 39.9 (11.0-144.4).3
gN=number of evaluable patients.
h95% CI: 48, 64.3
i95% CI: 0, 8.3
jReversal defined as mNIS+7 change from baseline of <0 points.
More than half of patients treated with ONPATTROexperienced reversal in neuropathy impairment from baseline3
- At 18 months, 56% of ONPATTRO-treated patients experienced reversal in neuropathy impairment compared to 4% of placebo-treated patients3
- For ONPATTRO-treated patients who did not experience reversal in neuropathy impairment, progression was slowed compared to the placebo arm (median change of 10 points vs 26 points)3
The benefit of ONPATTRO on neuropathy impairment was observed across all subgroups, including age, sex, mutation status, previous tetramer stabilizer use, and disease stage.1,3