WAINUA is indicated for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults.

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ABOUT hATTR

hATTR causes multiorgan dysfunction due to systemic deposition of amyloid fibrils1,2

hATTR is a protein-misfolding disorder of the transthyretin (TTR) protein1,2

The source of the disease is produced in the liver2,3

 

Liver

(where 90% of TTR
is produced)

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Transthyretin tetramer destabilizes

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Tetramer dissociates into monomers

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Monomers misfold and aggregate into amyloid fibrils

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Amyloid deposits throughout the body

Disease progression is rapid if left untreated4

The stages of progression5,6

hATTR-PN

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Walking without assistance

STAGE 1

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Walking with assistance

STAGE 2

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Confined to a wheelchair or bed

STAGE 3

LIFE EXPECTANCY IS 5 TO 15 YEARS FROM DIAGNOSIS7

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Explore the disease manifestations of hATTR

See how WAINUA targets TTR at the source

FOOTNOTES

hATTR, hereditary transthyretin-mediated amyloidosis; hATTR-PN, polyneuropathy of hereditary transthyretin-mediated amyloidosis.

REFERENCES

  1. 1. Nativi-Nicolau JN, Karam C, Khella S, Maurer MS. Screening for ATTR amyloidosis in the clinic: overlapping disorders, misdiagnosis, and multiorgan awareness. Heart Fail Rev. 2022;27(3):785-793.
  2. 2. Tschöpe C, Elsanhoury A. Treatment of transthyretin amyloid cardiomyopathy: the current options, the future, and the challenges. J Clin Med. 2022;11(8):2148.
  3. 3. Ioannou A, Fontana M, Gillmore JD. RNA targeting and gene editing strategies for transthyretin amyloidosis. BioDrugs. 2023;37(2):127-142.
  4. 4. Conceição I, González-Duarte A, Obici L, et al. "Red-flag"-symptom clusters in transthyretin familial amyloid polyneuropathy. J Peripher Nerv Syst. 2016;21(1):5-9.
  5. 5. Adams D, Ando Y, Beirão JM, et al. Expert consensus recommendations to improve diagnosis of ATTR amyloidosis with polyneuropathy. J Neurol. 2021;268(6):2109-2122.
  6. 6. Ando Y, Coelho T, Berk JL, et al. Guideline of transthyretin-related hereditary amyloidosis for clinicians. Orphanet J Rare Dis. 2013;8:31.
  7. 7. Hawkins PN, Ando Y, Dispenzeri A, Gonzalez-Duarte A, Adams D, Suhr OB. Evolving landscape in the management of transthyretin amyloidosis. Ann Med. 2015;47(8):625-638.

IMPORTANT SAFETY INFORMATION

WARNINGS AND PRECAUTIONS

  • Reduced Serum Vitamin A Levels and Recommended Supplementation WAINUA leads to a decrease in serum vitamin A levels. Supplement with recommended daily allowance of vitamin A. Refer patient to an ophthalmologist if ocular symptoms suggestive of vitamin A deficiency occur.

ADVERSE REACTIONS

Most common adverse reactions (≥9% in WAINUA-treated patients) were vitamin A decreased (15%) and vomiting (9%).

INDICATION

WAINUA injection, for subcutaneous use, 45 mg is indicated for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults.

 

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