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Additional Endpoints

Improvement in autonomic symptoms and ability to perform everyday activities1

Autonomic symptoms: 

  • Patients receiving ONPATTRO® (patisiran) experienced a significant benefit relative to placebo in reduction of autonomic symptoms

    —In ONPATTRO-treated patients, improvement compared to baseline was seen for the orthostatic intolerance and gastrointestinal domains, the 2 domains that contribute most to the overall COMPASS 31 score3

Change in COMPASS 31 score from baseline at 18 monthsc,d

ONPATTRO®-treated patients had a decrease in autonomic symptoms from baseline at 18 months

aN=number of patients assessed at 18 months. bCOMPASS 31 scores at baseline were 30.6 with ONPATTRO and 30.3 with placebo.1cONPATTRO-placebo treatment difference (LS mean): -7.53.4 d95% CI: -11.9, -3.2.4

CI=confidence interval; COMPASS 31=Composite Autonomic Symptom Score 31; LS=least squares.

Activities of daily living: 

Patients receiving ONPATTRO saw a significant benefit relative to placebo in the ability to perform activities of daily living and everyday function

Change in R-ODS score from baseline at 18 monthsc,d

ONPATTRO®-treated patients had improved ability to perform everyday activities compared to placebo from baseline at 18 months

aN=number of patients assessed at 18 months. bR-ODS scores at baseline were 29.7 with ONPATTRO and 29.8 with placebo.1cONPATTRO-placebo treatment difference (LS mean): 9.0.4 d95% CI: 7.0, 10.9.4

CI=confidence interval; R-ODS=Rasch-built Overall Disability Scale; LS=least squares.

ONPATTRO improved other key
measures of disease burden1,4,5

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Increased 

  • Gait speed from baseline at 18 months improved in ONPATTRO-treated
    patients by 0.08 m/sec     compared to placebo-treated patients whose speed worsened by -0.24 m/sec (p<0.001)a,b
  • 53% of patients treated with ONPATTRO showed an improvement in gait speed (change >0) at 18 months, compared to 13% of patients treated with placebo
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Improved 

  • ONPATTRO-treated patients maintained a better nutritional status at 18 months compared to placebo-treated patients as demonstrated by a 3.7-unit decrease in mBMI vs a 119-unit decease in the placebo group (p<0.001)c,d
  • Maintenance in mBMI vs placebo was seen in the ONPATTRO-treated patients as early as 3 months
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Improved motor strength

  • ONPATTRO-treated patients experienced a 0.1-point increase from baseline at 18 months in NIS-W score compared with the placebo-treated patients whose score increased by 17.9 points (p<0.001)e,f
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Improved patient mobility, self-care, usual activities, pain/discomfort, and anxiety/depression as measured by 6,7

  • ONPATTRO-treated patients showed improvement at 18 months in these domains of EQ-5D-5L compared to placebo-treated patients
  • EQ-5D-5L was an exploratory endpoint in the APOLLO study

aONPATTRO-placebo treatment difference (LS mean): 0.31.5 b95% CI: 0.23, 0.39.5 cONPATTRO-placebo treatment difference (LS mean): 116.5 d95% CI: 82, 149.5 eONPATTRO-placebo treatment difference (LS mean): -17.9.4 f95% CI: -22.3, -13.4.4
CI=confidence interval; EQ-5D-5L=Euro Quality of Life 5 Dimensions 5 Levels; LS=least squares; mBMI=modified body mass index; NIS-W=Neuropathy Impairment Score-Weakness.

NEXT: Learn about
the safety profile for ONPATTRO
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References:

  1. Adams D, Gonzalez-Duarte A, O’Riordan WD, et al. N Engl J Med. 2018;379(1):11-21.
  2. Adams D, Suhr OB, Dyck PJ, et al. BMC Neurol. 2017;17(1):181.
  3. Data on File CSR. Alnylam Pharmaceuticals, Inc. 2018. 
  4. Center for Drug Evaluation and Research. NDA 210922—patisiran—cross-discipline team leader review. U.S. Department of Health and Human Services, Food and Drug Administration; 2018.
  5. ONPATTRO [package insert]. Cambridge, MA: Alnylam Pharmaceuticals, Inc; 2018.
  6. Adams D, Gonzalez-Duarte A, O’Riordan W, et al. Slides presented at: American Academy of Neurology; April 25, 2018; Los Angleles, CA.
  7. Herdman M, Gudex C, Lloyd A, et al. Qual Life Res. 2011;20(10):1727-1736.
  8. Sletten DM, Suarez GA, Low PA, et al. Mayo Clin Proc. 2012;87(12):1196-1201.
  9. Berk K, Lin H, Agarwal S, et al. Poster presented at: XVIth International Symposium on Amyloidosis; March 26-29, 2018; Kumamoto, Japan.
  10. van Nes SI, Vanhoutte EK, van Doorn PA, et al. Neurology. 2011;76(4):337-345.
  11. Suhr O, Danielsson A, Holmgren G, et al. J Intern Med. 1994;235(5):479-485.