What is ATTR?

ATTR is a progressive and fatal disease1

Starting treatment at diagnosis is key to delaying disease progression2

ATTR is a multisystem disease caused by pathogenic TTR3-6

 

ATTR is a multisystem disease caused by pathogenic TTR3-6

Cardiomyopathy is a common manifestation of ATTR (ATTR-CM) that may lead to heart failure; musculoskeletal manifestations, polyneuropathy, and other symptoms may also present.4,6,12

  • Transthyretin-mediated amyloidosis (ATTR) is an underdiagnosed, progressive, debilitating, and fatal disease1
  • Patients with ATTR may be diagnosed 3 to 8 years after symptom onset1,13,14
  • If left untreated, the median survival for patients with ATTR is 2.5 to 5.5 years post-diagnosis15-18

There are 2 types of ATTR

Wild-type ATTR (wtATTR)

wtATTR is related to aging and typically manifests as cardiomyopathy. The etiology of wtATTR is unknown.10,19,20

Hereditary ATTR (hATTR)

hATTR is caused by a variant in the TTR gene and can manifest as cardiomyopathy or polyneuropathy. Mixed phenotype is the most common presentation.10,19,20

Either type of ATTR can have a heterogeneous symptom presentation.10

ATTR is a multisystem disease10

The presence of these red-flag symptoms may help identify patients with ATTR

Cardiac

Elevated NT-proBNP, unexplained left ventricular wall thickening in the absence of hypertension, conduction system disease/atrial fibrillation, HFpEF in combination with other noncardiac red-flag symptoms21-26

Sensory-motor neuropathy

Length-dependent neuropathic pain and weakness, altered sensation, difficulty walking2

Gastrointestinal manifestations

Diarrhea, constipation, nausea, vomiting, unintentional weight loss2

Musculoskeletal

History of musculoskeletal symptoms (eg, carpal tunnel syndrome, lumbar spinal stenosis, or biceps tendon rupture), past orthopedic surgery21,23

Autonomic neuropathy

Orthostatic hypotension, recurrent urinary tract infections, sexual dysfunction, sweating abnormalities, urinary retention2

 

AMVUTTRA does not treat all of the symptoms of ATTR. AMVUTTRA is approved to treat ATTR-CM and hATTR-PN in adults.

 

ATTR-CM=cardiomyopathy of transthyretin-mediated amyloidosis; hATTR-PN=polyneuropathy of hereditary transthyretin-mediated amyloidosis; HFpEF=heart failure with preserved ejection fraction; NT‑proBNP=N-terminal prohormone of brain-type natriuretic peptide.

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Up next: See how AMVUTTRA works
TTR=transthyretin. 
 
 

Important Safety Information

Reduced Serum Vitamin A Levels and Recommended Supplementation

AMVUTTRA 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

In a study of patients with hATTR-PN, 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%).

In a study of patients with ATTR-CM, no new safety issues were identified.

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

Indications

AMVUTTRA® (vutrisiran) is indicated for the treatment of the:

  • cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM) in adults to reduce cardiovascular mortality, cardiovascular hospitalizations and urgent heart failure visits.
  • polyneuropathy of hereditary transthyretin-mediated amyloidosis (hATTR-PN) in adults.

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

References

  1. Hawkins et al. Ann Med. 2015;47(8):625–638.
  2. Conceição et al. Amyloid. 2019;26(1):3–9.
  3. Hanna. Curr Heart Fail Rep. 2014;11(1):50–57.
  4. Mohty et al. Arch Cardiovasc Dis. 2013;106(10):528–540.
  5. Adams et al. Neurology. 2015;85(8):675–682.
  6. Koike & Katsuno. Biomedicines. 2019;7(1):11.
  7. Soprano et al. J Biol Chem. 1985;260(21):11793-11798.
  8. Holmgren et al. Clin Genet. 1991;40(3):242-246.
  9. Bezerra et al. Front Mol Neurosci. 2020;13:592644.
  10. Nativi-Nicolau et al. Heart Fail Rev. 2022;27(3):785-793.
  11. Lane et al. Circulation. 2019;140(1):16–26.
  12. Coelho et al. Curr Med Res Opin. 2013;29(1):63-76.
  13. Adams et al. J Neurol. 2021;268(6):2109-2122.
  14. Rozenbaum et al. Cardiol Ther. 2021;10(1):141-159.
  15. Aus dem Siepen et al. Clin Res Cardiol. 2018;107(2):158-169.
  16. Gertz et al. Mayo Clin Proc. 1992;67(5):428-440.
  17. Swiecicki et al. Amyloid. 2015;22(2):123-131.
  18. Givens et al. Aging Health. 2013;9(2):229-235.
  19. Ando et al. Orphanet J Rare Dis. 2013;8(1):31.  
  20. Kourelis & Gertz. Expert Rev Cardiovasc Ther. 2015;13(8):945-961.
  21. Maurer et al. Circ Heart Fail. 2019;12(9):e006075.
  22. Kittleson et al. Circulation. 2020;142(1):e7–22.
  23. Kittleson et al. J Am Coll Cardiol. 2023;81(11):1076-1126. 
  24. Dharmarajan K, Maurer MS. J Am Geriatr Soc. 2012;60(4):765-774. 
  25. Brito et al. Global Heart. 2023;18(1):59.
  26. González-López et al. Eur Heart J. 2015;36(38):2585-2594.