What is Wainua medication?

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Wainua (Eplontersen): An Antisense Oligonucleotide for Hereditary ATTR Polyneuropathy

Wainua (eplontersen) is a ligand-conjugated antisense oligonucleotide approved by the FDA in December 2023 for treating polyneuropathy caused by hereditary transthyretin-mediated amyloidosis (ATTRv-PN). 1

Mechanism of Action

  • Eplontersen is targeted to the liver through a ligand containing three N-acetyl galactosamine residues, where it binds to both wild-type and variant TTR mRNA. 1
  • This binding reduces circulating transthyretin (TTR) protein levels and decreases amyloid deposition in tissues. 1
  • The drug works by preventing the production of the abnormal protein that causes nerve damage in hereditary ATTR amyloidosis. 1

Clinical Efficacy

  • In phase III trials, subcutaneous eplontersen reduced serum TTR levels, inhibited neuropathy progression, and improved health-related quality of life in patients with ATTRv-PN. 1
  • These results formed the basis for FDA approval for treating polyneuropathy in hereditary ATTR amyloidosis. 1

Administration and Development Status

  • Eplontersen is administered as a subcutaneous injection. 1
  • The medication is currently undergoing regulatory review in the EU, UK, Switzerland, and Canada for similar indications. 1
  • Phase III development is ongoing for ATTR cardiomyopathy, expanding potential therapeutic applications beyond polyneuropathy. 1

Manufacturer and Development

  • Eplontersen is being developed jointly by Ionis Pharmaceuticals and AstraZeneca. 1
  • The drug represents a targeted approach to treating the underlying genetic cause of hereditary ATTR amyloidosis rather than just managing symptoms. 1

References

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This information is intended for healthcare professionals. Any medical decision-making should rely on clinical judgment and independently verified information. The content provided herein does not replace professional discretion and should be considered supplementary to established clinical guidelines. Healthcare providers should verify all information against primary literature and current practice standards before application in patient care. Dr.Oracle assumes no liability for clinical decisions based on this content.

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