Medicines
Discover all available medicines and treatments
Discover all available medicines and treatments
Brand: Kynamro
Mipomersen is an antisense oligonucleotide drug used to treat homozygous familial hypercholesterolemia (HoFH), a severe genetic disorder characterized by extremely high levels of low-density lipoprotein (LDL) cholesterol. It works by inhibiting the production of apolipoprotein B-100 (apoB-100), a protein essential for the formation and transport of LDL cholesterol. Mipomersen belongs to the class of lipid-lowering agents.
For the treatment of homozygous familial hypercholesterolemia (HoFH) in conjunction with other lipid-lowering therapies and dietary modifications.
Mipomersen can cause liver toxicity, including increases in liver fat and liver enzyme elevations. Patients should be monitored for liver function before and during treatment.
Outcome:
Increased risk of myopathy and rhabdomyolysis
Mechanism:
Additive effects on muscle toxicity
Outcome:
Increased risk of liver toxicity
Mechanism:
Additive effects on liver function
Outcome:
May decrease mipomersen absorption
Mechanism:
Changes in gastric pH
Most likely new formulation: Sustained-release injection (Year 2026, 40% confidence)
Based on current usage patterns and safety profile, mipomersen is likely to remain a niche therapy for HoFH with limited market expansion (60% confidence).
Antisense Oligonucleotide, Lipid-Lowering Agent
Oligonucleotide