- 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
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
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
- 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
- Maintenance in mBMI vs placebo was seen in the ONPATTRO-treated patients as early as 3 months
Improved motor strength
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.