Efficacy

AMVUTTRA™ significantly improved polyneuropathy1,2

Primary endpoint: change from baseline at 9 months in mNIS+71

  • mNIS+7 is a composite measure that assesses motor strength, reflexes, sensation, nerve conduction, and postural blood pressure (score range 0 to 304)1
Change from baseline in mNIS+71,2,a-c
Change in mNIS+7 from baseline at 18 months with AMVUTTRA™ (vutrisiran) versus external placebo

aMean mNIS+7 at baseline was 60.6 with AMVUTTRA and 74.6 with external placebo group.1

bBars represent SEM.

cN=number of evaluable patients.

dReversal defined as mNIS+7 change from baseline of <0 points.

CI=confidence interval; LS=least squares.

48% of patients treated with AMVUTTRA experienced reversald in neuropathy impairment from baseline at 18 months, compared with 4% of patients in the external placebo group.2,3

aMean mNIS+7 at baseline was 60.6 with AMVUTTRA and 74.6 with external placebo group.1

bBars represent SEM.

cN=number of evaluable patients.

dReversal defined as mNIS+7 change from baseline of <0 points.

CI=confidence interval; LS=least squares.

AMVUTTRA significantly improved quality of life2

AMVUTTRA helped to alleviate the burden of the disease, as measured by Norfolk QoL-DN1,2

  • Norfolk QoL-DN is a patient-reported assessment that evaluates the effects of neuropathy in domains such as physical functioning, activities of daily living, symptoms, and autonomic function (score range -4 to 136)1
Change from baseline in Norfolk QoL-DN score2,a-c
Change in Norfolk QoL-DN from baseline at 18 months with AMVUTTRA™ (vutrisiran) versus external placebo

aNorfolk QoL-DN scores at baseline were 47.1 with AMVUTTRA and 55.5 with external placebo group.1

bBars represent SEM. 

cN=number of evaluable patients.

57% of patients treated with AMVUTTRA experienced improvement in quality of life from baseline at 18 months, compared with 10% of patients in the external placebo group.2

aNorfolk QoL-DN scores at baseline were 47.1 with AMVUTTRA and 55.5 with external placebo group.1

bBars represent SEM. 

cN=number of evaluable patients.

AMVUTTRA improved other key measures of disease burden
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  • AMVUTTRA-treated patients experienced significant improvement in nutritional status from baseline at 18 months compared with placebo-treated patients, who experienced a decline (25.0 units, -115.7 units, respectively [p<0.001])2
    • Improvement in mBMI vs placebo was observed after 1 dose of AMVUTTRA3
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  • Patients treated with AMVUTTRA maintained a better gait speed from baseline at 18 months compared with those treated with placebo (-0.024 m/sec, -0.264 m/sec, respectively [p<0.001])2
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  • R-ODS is a 24-item scale that evaluates limitations on everyday activities such as showering, going shopping, carrying a heavy object, walking up stairs, and/or walking outdoors (score range 0 to 48)4
  • AMVUTTRA-treated patients were better able to perform activities of daily living compared with placebo-treated patients (LS mean change from baseline at 18 months was -1.5 and -9.9, respectively [p<0.001])2

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. Adams D, Tournev I, Taylor M, et al. Slides presented at la Société Francophone du Nerf Périphérique (SFNP); January 21-22, 2022.
  3. Data on file. Alnylam Pharmaceuticals, Inc.
  4. van Nes SI, Vanhoutte EK, van Doorn PA, et al. Neurology. 2011;76(4):337-345.

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.