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

Kelly sits on the front stairs of her house.
Kelly sits on the front stairs of her house.
Disease characteristics of patient populationPatients (%)

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.