Study Design

The efficacy and safety of AMVUTTRA® were evaluated in HELIOS-A

Global, randomized, open-label, multicenter, Phase 3 study1-3

HELIOS-A Phase 3 study design
HELIOS-A Phase 3 study design
  • 164 patients with the polyneuropathy of hATTR amyloidosis were randomized 3:1 to the AMVUTTRA group or the patisiran reference group1,2
  • Efficacy assessments were based on a comparison of the AMVUTTRA arm of the study with an external placebo group from APOLLO (n=77), a randomized controlled study in a comparable patient population with polyneuropathy caused by hATTR amyloidosis1,2,a
  • The patisiran reference group was included in the study to validate the use of the external placebo group3
  • 96% of AMVUTTRA-treated patients completed at least 18 months of treatment2

aStudy patients were ≥18 years of age, had a diagnosis of hATTR amyloidosis with polyneuropathy caused by any TTR variant, a Polyneuropathy Disability (PND) score ≤IIIb, a Neuropathy Impairment Score (NIS) of 5–130, a Karnofsky Performance Status (KPS) score ≥60%, and were permitted to have previously used TTR stabilizers.2

BMI=body mass index; hATTR=hereditary transthyretin-mediated; IV=intravenous; mNIS+7=modified Neuropathy Impairment Score + 7; Norfolk QoL-DN=Norfolk Quality of Life-Diabetic Neuropathy; q3m=every 3 months; q3w=every 3 weeks; SC=subcutaneous.

HELIOS-A included a diverse patient population

Participants in the study were representative of the real-world population of patients with the polyneuropathy of hATTR amyloidosis1-3

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Global study

164 patients from 22 countries2

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Broad age range

26 to 85 years old (median: 60 years old)2,3

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Multiple variants

26 different TTR variants were represented

V30M (45%), T60A (11%), E89Q (9%), A97S (5%), V122I (4%), S50R (3%), S77Y (3%), and D38A (3%)2,3

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Prior treatment

66% of patients had previously been treated with tafamidis or diflunisal2,3

Gary, treated with AMVUTTRA® (vutrisiran), sitting at a table in a park on a sunny day.
Gary, treated with AMVUTTRA® (vutrisiran), sitting at a table in a park on a sunny day.
Disease characteristics of patient population Patients (%)

Stage I polyneuropathy

Unimpaired ambulation (mostly mild sensory and motor neuropathy in lower limbs)

 70%3

Stage II polyneuropathy

Assistance with ambulation required (mostly moderate impairment progression to the lower limbs, upper limbs, and trunk)

 30%3
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Important Safety Information and Indication

Important Safety Information

Reduced Serum Vitamin A Levels and Recommended Supplementation

AMVUTTRA® (vutrisiran) treatment leads to a decrease in serum vitamin A levels.

Supplementation at the recommended daily allowance (RDA) of vitamin A is advised for patients taking AMVUTTRA. Higher doses than the RDA should not be given to try to achieve normal serum vitamin A levels during treatment with AMVUTTRA, as serum vitamin A levels do not reflect the total vitamin A in the body.

Patients should be referred to an ophthalmologist if they develop ocular symptoms suggestive of vitamin A deficiency (e.g., night blindness).

Adverse Reactions

The most common adverse reactions that occurred in patients treated with AMVUTTRA were pain in extremity (15%), arthralgia (11%), dyspnea (7%), and vitamin A decreased (7%).

Indication

AMVUTTRA is indicated for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults.

For additional information about AMVUTTRA, please see the full Prescribing Information.

References

  1. AMVUTTRA Prescribing Information. Cambridge, MA: Alnylam Pharmaceuticals, Inc.
  2. Adams D, Tournev IL, Taylor MS, et al. Amyloid. 2023;30(1):18-26.
  3. Data on file. Alnylam Pharmaceuticals, Inc.