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:

Sensory-motor neuropathy
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E.g., length-dependent neuropathic pain and weakness, altered sensation, difficulty walking, bilateral carpal tunnel syndrome

Autonomic neuropathy
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E.g., orthostatic hypotension, recurrent urinary tract infections, sexual dysfunction, sweating abnormalities, urinary retention

Gastrointestinal manifestations
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E.g., diarrhea, constipation, nausea, vomiting, unintentional weight loss

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

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Up next Learn about AMVUTTRA

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. Ando Y, Coelho T, Berk JL, et al. Orphanet J Rare Dis. 2013;8:31.
  2. Coutinho P, Martins da Silva A, Lopes Lima J, Resende Barbosa A. Excerpta Medica. 1980:88-98.
  3. Conceição I, Gonzalez-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.