Synonyms: ISIS 301012 parent acid | Kynamro®
mipomersen is an approved drug (FDA (2013))
Compound class:
Synthetic organic
Comment: MOE in the nucleotide image denotes nucleosides with 2'-O-(2-methoxyethyl) modification. The drug formulation contains the sodium salt, mipomersen sodium (CHEMBL2107849).
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No information available. |
Summary of Clinical Use |
Used as an adjunct to diet and other lipid-lowing medications in the treatment of homozygous familial hypercholesterolemia. |
Mechanism Of Action and Pharmacodynamic Effects |
Mipomersen is a cholesterol-reducing antisense therapeutic. It targets the messenger RNA for apolipoprotein B. Mipomersen binds to the mRNA of one of apolipoprotein B's isoforms, Apo B-100 (Uniprot P04114). The resulting double-stranded RNA is degraded by RNase H, preventing translation of the mRNA and formation of the apo B-100 protein. Mipomersen binds at nucleotide positions 3249-3268 relative to the published sequence provided in GenBank accession number NM_000384. |
External links |
For extended ADME data see the following: Drugs.com |