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Study Design

APOLLO—the largest controlled study in hereditary transthyretin-mediated (hATTR) amyloidosis1

Global, randomized, double-blind, multicenter, placebo-controlled study of 225 patients
over 18 months1-3

Phase 3 trial in adult patients with the polyneuropathy of hATTR amyloidosis

93% of patients treated with ONPATTRO® (patisiran) and 62% of patients treated with placebo completed 18 months of the assigned treatment2

aStudy patients had a diagnosis of hATTR amyloidosis with polyneuropathy caused by any transthyretin (TTR) mutation, had FAP stage 1 or 2 disease, had a Neuropathy Impairment Score (NIS) of 5-130, and were permitted to have previously used tetramer stabilizers.

10MWT=10-meter walk test; COMPASS 31=Composite Autonomic Symptom Score 31; EQ-5D-5L=EuroQoL 5 Dimensions 5 Levels; FAP=familial amyloid polyneuropathy; mBMI=modified body mass index; mNIS+7=modified Neuropathy Impairment Score + 7; NIS-W=Neuropathy Impairment Score-Weakness; Norfolk QoL-DN=Norfolk Quality of Life-Diabetic Neuropathy; R-ODS=Rasch-built Overall Disability Scale.

When patients completed their participation
in the 18-month APOLLO study,

99%b of eligible patients

elected to enter the open-label extension study.3

b186 of 187 patients.

The APOLLO study included a diverse patient population

  • Global study1,3

    225 patients from 19 countries

  • Broad age range3

    24 to 83 years old (median 62 years)

  • Multiple mutations2,3

    39 different TTR mutations were represented in the study population

  • Prior treatment3

    53% of patients had previously been treated with tafamidis or diflunisal

Disease characteristics of
patient population2,c

46%

Stage 1 polyneuropathy2

Unimpaired ambulation

(mostly mild sensory, motor, and
autonomic neuropathy in lower limbs)

53%

Stage 2 polyneuropathy2

Assistance with ambulation required

(mostly moderate impairment progression
to the lower limbs, upper limbs, and trunk)

cFAP stage percentages do not add up to 100% due to inclusion of 1 stage 3 patient.3

Participants in APOLLO reflect
real-world patient
demographics, demonstrating the potential for ONPATTRO
to treat polyneuropathy manifestations in a variety of adults with hATTR amyloidosis.3

NEXT: See how ONPATTRO
improved polyneuropathy
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References:

  1. Adams D, Suhr OB, Dyck PJ, et al. BMC Neurol. 2017;17(1):181.
  2. ONPATTRO [package insert]. Cambridge, MA: Alnylam Pharmaceuticals, Inc; 2018.
  3. Adams D, Gonzalez-Duarte A, O’Riordan WD, et al. N Engl J Med. 2018;379(1):11-21.