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mipomersen
- 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
Mipomersen is a second-generation antisense oligonucleotide used as an adjunct to diet and other lipid-lowering medications for the treatment of homozygous familial hypercholesterolemia (HoFH), a rare genetic disorder characterized by extremely high cholesterol levels. It works by binding specifically to the messenger RNA (mRNA) encoding apolipoprotein B-100 (apoB-100), the main protein component of low-density lipoprotein cholesterol (LDL-C) and very low-density lipoprotein cholesterol (VLDL-C). This binding leads to RNase H-mediated degradation of apoB-100 mRNA, thereby inhibiting translation and reducing production of apoB-containing lipoproteins, including LDL-C, VLDL-C, and Lipoprotein(a). Mipomersen is administered via subcutaneous injection. The drug was developed by Ionis Pharmaceuticals and marketed under the brand name Kynamro. Due to risks of hepatotoxicity, its use is restricted under a Risk Evaluation and Mitigation Strategy program[2][3][5][6][7].
- Brand names
- Kynamro
- Other names
- mipomersen sodium
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