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Endothelins (ETs) are potent vasoactive peptides critical in the human body's various physiological and pathological processes. One of the most promising therapeutic strategies for treating pulmonary arterial hypertension (PAH) involves counteracting the effects of these endothelins using a class of drugs known as endothelin receptor antagonists.
ETs are synthesized through a complex sequence of enzymatic steps, primarily involving an enzyme referred to as endothelin-converting enzyme...
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Eplontersen: First Approval.

Tina Nie1

  • 1Springer Nature, Private Bag 65901, Mairangi Bay, Auckland, 0754, New Zealand. dru@adis.com.

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|February 27, 2024
PubMed
Summary

Eplontersen significantly reduced TTR protein levels and slowed neuropathy progression in hereditary transthyretin-mediated amyloidosis (hATTR-PN). This led to its recent US approval for treating this rare disease.

Area of Science:

  • Pharmacology
  • Genetics
  • Neurology

Background:

  • Transthyretin-mediated amyloidosis (ATTR) is a progressive disease caused by misfolded transthyretin (TTR) protein.
  • ATTR leads to amyloid deposition in nerves and organs, causing significant morbidity and mortality.
  • Current treatments are limited, highlighting the need for novel therapeutic approaches.

Purpose of the Study:

  • To summarize the development of eplontersen, a novel antisense oligonucleotide for treating ATTR.
  • To highlight the key milestones leading to the regulatory approval of eplontersen for ATTRv-PN.

Main Methods:

  • Eplontersen is a ligand-conjugated antisense oligonucleotide targeting TTR mRNA in the liver.
  • It utilizes N-acetyl galactosamine residues for targeted delivery.

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  • Phase III clinical trials evaluated its efficacy and safety in patients with ATTRv-PN.

    • Main Results:

    • Subcutaneous eplontersen demonstrated a reduction in serum TTR levels.
    • The treatment inhibited the progression of neuropathy in patients with ATTRv-PN.
    • Health-related quality of life showed improvement in treated patients.

    Conclusions:

    • Eplontersen represents a significant advancement in the treatment of ATTRv-PN.
    • The drug's targeted mechanism effectively reduces TTR protein and amyloid burden.
    • Regulatory approvals in the US and ongoing reviews in other regions signify its therapeutic potential.