Study Design

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

Global, randomized, open-label, multicenter, Phase 3 study1,2

Global, randomized, open-label, multicenter, Phase 3 study1,2
HELIOS-A Phase 3 study design

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.3

BMI=body mass index; IV=intravenous; q3m=every 3 months; q3w=every 3 weeks; SC=subcutaneous.

  • 164 patients with the polyneuropathy of hATTR amyloidosis were randomized 3:1 to the AMVUTTRA group or the patisiran reference group1
  • 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 population1,a
  • The patisiran reference group was included in the study to validate the use of the external placebo group2
  • 96% of AMVUTTRA-treated patients completed at least 18 months of treatment2

Participants in the study were representative of the real-world population of patients with the polyneuropathy of hATTR amyloidosis.1,2

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.3

BMI=body mass index; IV=intravenous; q3m=every 3 months; q3w=every 3 weeks; SC=subcutaneous.

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 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. Data on file. Alnylam Pharmaceuticals, Inc.
  3. Adams D, Tournev I, Taylor M, et al. Slides presented at American Academy of Neurology; April 17-22, 2021. 

Important Safety Information and Indication

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 arthralgia (11%), dyspnea (7%), and vitamin A decreased (7%).

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

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 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.