Disease Overview

Hereditary ATTR amyloidosis: a multisystem disease with frequent and early polyneuropathy manifestations1-5

Hereditary transthyretin-mediated (hATTR) amyloidosis is caused by a variant in the transthyretin (TTR) gene that results in the accumulation of amyloid deposits in multiple sites in the body.1,3

Early symptoms of hATTR amyloidosis may include3:

hATTR amyloidosis has a heterogeneous symptom presentation. Other symptoms that may raise clinical suspicion include3,6:

Cardiovascular manifestations

E.g., arrhythmias, conduction abnormalities, heart failure

Renal abnormalities

E.g., renal impairment, cardiorenal syndrome

Ocular involvement

E.g., vitreous opacity, glaucoma

AMVUTTRA™ does not treat all of the symptoms of hATTR amyloidosis. AMVUTTRA is indicated for the treatment of the polyneuropathy of hATTR amyloidosis in adults.7

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. Ando Y, Coelho T, Berk JL, et al. Orphanet J Rare Dis. 2013;8:31.
  2. Coutinho P, Martins da Silva A, Lopes Lima JL, et al. Excerpta Medica. 1980:88-98.
  3. Conceição I, González-Duarte A, Obici L, et al. J Peripher Nerv Syst. 2016;21(1):5-9.
  4. Ando Y, Nakamura M, Araki S. Arch Neurol. 2005;62(7):1057-1062.
  5. Coelho T, Merlini G, Bulawa CE, et al. Neurol Ther. 2016;5(1):1-25.
  6. Ruberg FL, Grogan M, Hanna M, et al. J Am Coll Cardiol. 2019;73(22):2872-2891.
  7. AMVUTTRA Prescribing Information. Cambridge, MA: Alnylam Pharmaceuticals, Inc.

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.