How AMVUTTRA works

How AMVUTTRA® Works

Suppress TTR production at the source with AMVUTTRA1

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See how treatment with AMVUTTRA led to a rapid knockdown of TTR protein in serum.

AMVUTTRA addresses the underlying cause of ATTR-CM and hATTR-PN with rapid knockdown of TTR1-3

 

AMVUTTRA addresses the underlying cause of ATTR-CM and hATTR-PN with rapid knockdown of TTR1,3

Intervene early with AMVUTTRA, the first and only silencer approved for ATTR‑CM and hATTR‑PN1,21,22

  • AMVUTTRA is formulated for targeted delivery to the liver, the primary source of TTR production1,2
  • AMVUTTRA deploys the body’s natural silencing complex to act upstream of tetramer formation1,20

mRNA=messenger RNA; RNAi=ribonucleic acid interference; TTR=transthyretin.

AMVUTTRA delivered RAPID KNOCKDOWN of TTR as early as 6 weeks in HELIOS-B3,23*

Knockdown of TTR was sustained through 30 months23†‡

Chart showing the median TTR trough reduction at 30 monthsChart showing the median TTR trough reduction at 30 months
 
 

Knockdown of TTR was sustained through 30 months23†‡

Chart showing the median TTR trough reduction at 30 months

  • In the HELIOS-B pivotal trial, serum TTR was evaluated in patients with ATTR-CM treated with 25 mg of AMVUTTRA via subcutaneous injection once every 3 months1
  • A similar reduction in TTR levels was observed regardless of baseline tafamidis use, disease type (wtATTR or hATTR), sex, age, weight, or race1

*TTR knockdown was first measured in the serum at 6 weeks in HELIOS-B 23

†TTR knockdown level is demonstrated through serum TTR reduction. 1

‡Bars indicate 95% confidence intervals. 23

CI=confidence interval; hATTR=hereditary ATTR; TTR=transthyretin; wtATTR=wild-type ATTR.

AMVUTTRA achieves sustained knockdown of TTR with a subcutaneous injection 4 times per year.1

Treatment with AMVUTTRA led to rapid knockdown of TTR as early as 3 weeks in HELIOS-A24,a

Knockdown of TTR was sustained over 18 months24,25,b,c

Chart showing mean TTR knockdown as high as 88%Chart showing mean TTR knockdown as high as 88%
 

Knockdown of TTR was sustained over 18 months24,25,b,c

aFirst measured at 3 weeks.

bBars represent SEM.

cTTR knockdown level is demonstrated through serum TTR reduction.

KD=knockdown; SEM=standard error of the mean.

  • Serum TTR was evaluated in patients with hATTR-PN treated with 25 mg AMVUTTRA via subcutaneous injection once every 3 months1
  • With AMVUTTRA, similar rapid knockdown in serum TTR was observed regardless of V30M variant status, previous TTR stabilizer use, sex, age, weight, or race1,23

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Up next: HELIOS-B study design
Up next: HELIOS-A study design

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. AMVUTTRA Prescribing Information. Cambridge, MA: Alnylam Pharmaceuticals, Inc.
  2. Bezerra et al. Front Mol Neurosci. 2020;13:592644.
  3. Fontana et al. N Engl J Med. 2025;392(1):33-44.
  4. Hanna. Curr Heart Fail Rep. 2014;11(1):50–57.
  5. Mohty et al. Arch Cardiovasc Dis. 2013;106(10):528–540.
  6. Adams et al. Neurology. 2015;85(8):675–682.
  7. Koike & Katsuno. Biomedicines. 2019;7(1):11. 
  8. Soprano et al. J Biol Chem. 1985;260(21):11793-11798.
  9. Holmgren et al. Clin Genet. 1991;40(3):242-246.
  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. Hawkins et al. Ann Med. 2015;47(8):625–638.
  14. Adams et al. J Neurol. 2021;268(6):2109-2122. 
  15. Rozenbaum et al. Cardiol Ther. 2021;10(1):141-159.
  16. Aus dem Siepen et al. Clin Res Cardiol. 2018;107(2):158-169.
  17. Gertz et al. Mayo Clin Proc. 1992;67(5):428-440.
  18. Swiecicki et al. Amyloid. 2015;22(2):123-131.
  19. Givens et al. Aging Health. 2013;9(2):229-235.
  20. Habtemariam et al. Clin Pharmacol Ther. 2021;109(2):372-382.
  21. Tomasoni et al. Front Cardiovasc Med. 2023;10:1154594.
  22. Williams et al. J Cardiothorac Vasc Anesth. 2024;38(7):1457-1459.
  23. Data on file. Alnylam Pharmaceuticals, Inc.
  24. Adams et al. Amyloid. 2023;30(1):18-26. 
  25. Adams et al. N Engl J Med. 2018;379(1):11-21.