Patisiran for the treatment of hereditary transthyretin-mediated amyloidosis

ABSTRACT

Introduction: Hereditary transthyretin-mediated amyloidosis is caused by a mutation in transthyretin (TTR) gene resulting in misfolded TTR protein accumulating as amyloid fibrils. Patisiran is a lipid nanoparticle formulation of ribonucleic acid interference (RNAi), which can reduce the production of TTR.

Areas covered: In this review, the chemical property, mechanism of action, pharmacokinetics, clinical efficacy, and safety of patisiran were introduced.

Expert Commentary: Patisiran offers a new treatment option for patients with hereditary transthyretin-mediated amyloidosis. Patisiran can significantly reduce the TTR level and improve patient’s neuropathy and quality of life. The common adverse reactions were upper respiratory tract infections and infusion-related reactions.

KEYWORDS:

• Hereditary transthyretin-mediated amylodiosis
• infusion-related reactions
• patisiran
• ribonucleic acid interference
• transthyretin

Declaration of interest

The author has no relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript. This includes employment, consultancies, honoraria, stock ownership or options, expert testimony, grants or patents received or pending, or royalties.

Reviewer disclosures

Peer reviewers on this manuscript have no relevant financial or other relationships to disclose.

Alnylam Pharmaceuticals, Inc. provided a scientific accuracy review at the request of the journal editor.

Additional information

Funding

This manuscript was not funded.