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Quality of Life

ONPATTRO® (patisiran) significantly improved quality of life1

Key Secondary Endpoint: 1,2

  • Change from baseline at 18 months vs placebo in Norfolk Quality of Life-Diabetic Neuropathy (QoL-DN)
  • Norfolk QoL-DN score is a patient-reported assessment that evaluates neuropathy in domains such as physical function, activities of daily living, symptoms, and autonomic neuropathy (score range -4 to 136)

Change in Norfolk QoL-DN score1,a-c

Change in Norfolk QoL-DN score from baseline at 18 months

aNorfolk QoL-DN scores at baseline were 59.6 with ONPATTRO and 55.5 with placebo.1 bBars represent SEM. cN=number of evaluable patients. d95% CI: -19.8, -10.2.1 e95% CI: -27.2, -15.0.1

CI=confidence interval; LS=least squares; SEM=standard error of the mean.

Improvement in quality of life from baseline3,f,g

51% of ONPATTRO® (patisiran)-treated patients experienced improvement in QoL from baseline at 18 months vs 10% of placebo-treated patients

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: 10.0 (4.4-22.5).3
gPercentages based on mITT population: ONPATTRO (n=148); placebo (n=77).1
h95% CI: 43, 59.3
i95% CI: 4, 17.3

mITT=modified Intention-To-Treat.

More than half of patients treated with ONPATTRO experienced improvement in quality of life3

  • 51% of patients treated with ONPATTRO experienced improvement in quality of life compared to 10% of patients treated with placebo
  • Improvements relative to placebo were observed across all domains of Norfolk QoL-DN

The benefit of ONPATTRO on quality of life was observed across all subgroups, including age, sex, V30M mutation status, previous tetramer stabilizer use, and disease stage.

ONPATTRO demonstrated significant improvement in quality of life as measured by physical, functional, and social assessments.1,4

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References:

  1. ONPATTRO [package insert]. Cambridge, MA: Alnylam Pharmaceuticals, Inc; 2018.
  2. Vinik EJ, Vinik AI, Paulson JF, et al. J Peripher Nerv Syst. 2014;19:104-119.
  3. Adams D, Gonzalez-Duarte A, O’Riordan WD, et al. N Engl J Med. 2018;279(1):11-21.
  4. Vinik EJ, Hayes RP, Oglesby A, et al. Diabetes Technol Ther. 2005;7(3):497-508.