Drug intelligence / Profile preview

volanesorsen

Development stage
Phase 3
Lead developer
Ionis Pharmaceuticals
Modality
MicroRNA (miRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
Administration
Subcutaneous
01

Overview

Volanesorsen is a second-generation 2'-O-methoxyethyl (2'-MOE) chimeric antisense oligonucleotide designed to reduce triglyceride levels by targeting the messenger RNA (mRNA) for apolipoprotein C-III (apoC-III). By binding to the apoC-III mRNA, volanesorsen induces its degradation via RNase H1-mediated cleavage, thereby preventing translation of the apoC-III protein. ApoC-III normally inhibits triglyceride metabolism and hepatic clearance of chylomicrons; thus, inhibition of its production enables enhanced breakdown and clearance of triglycerides through both lipoprotein lipase-dependent and -independent pathways. Volanesorsen is primarily indicated as an adjunct to diet in adults with genetically confirmed familial chylomicronemia syndrome (FCS) who are at high risk for pancreatitis and have not responded adequately to other triglyceride-lowering therapies. The drug was discovered and developed by Ionis Pharmaceuticals[1][2][6].

Brand names
Waylivra
Other names
antisense oligonucleotide against apoCIIIAPOC3 antisense oligonucleotideAPOC-3 antisense oligonucleotideAPOC 3 antisense oligonucleotideApoC-III inhibitor
02

Targets

APOC3 (Apolipoprotein C-III)

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