DB05528ApprovedGene TherapiesAntisense OligonucleotidesApolipoprotein B-100 Synthesis InhibitorHypolipidemic Agents Indicated for Hyperlipidemia
Mipomersen is an oligonucleotide drug used for the treatment of homozygous familial hypercholesterolemia. Mipomersen sodium, which was known as the investigational drug, isis-301012, is the salt form of a synthetic phosphorothioate oligonucleotide.
- Mechanism
- Curator reviewed
Mipomersen binds to the mRNA that codes for apoB-100. This binding leads to double-stranded RNA, which is degraded by RNase H and prevents translation of the mRNA to form the apo B-100 protein.
- Primary indication
- Used in patients with homozygous familial hypercholesterolemia as an adjunct to diet and other lipid-lowering medications.Curator reviewed · 1 structured indication
- First approval
- United States, 2013
- Code names
- ISIS-301012
- Brand names
Resolves to
Clinical / RWD
Targets
What you can answer from here — as of September 13, 2026
2Protein targetsEach mapped to UniProt, with action and pharmacological actionListed above
Which drugs share a target with Mipomersen, and which of those have an active Phase 3 trial?
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