Clinical trials of mipomersen have primarily focused on patients with homozygous familial hypercholesterolemia (HoFH). However, studies have also explored its use in other conditions, including severe hypercholesterolemia, as an adjunct to statin therapy, and in heterozygous familial hypercholesterolemia (HeFH).

Mipomersen has consistently demonstrated significant reductions in LDL-C, Lp(a), apo-B, and total non-HDL cholesterol compared to placebo across various patient populations. Despite promising efficacy, its use outside of HoFH is discouraged due to risks of hepatotoxicity and limited evidence supporting cardiovascular benefits.

Mipomersen is currently FDA-approved exclusively for patients with homozygous familial hypercholesterolemia (HoFH). Further research is necessary to evaluate its potential as an adjunct to statin therapy in patients without HoFH. Notably, mipomersen is not included in recent AHA/ACC guidelines.

Meta-analyses indicate that mipomersen treatment may lead to various adverse effects, including injection site reactions, liver enzyme elevation, hepatic steatosis, and flu-like symptoms. These symptoms contribute to a higher rate of treatment discontinuation among patients receiving mipomersen.

As a newer cholesterol-lowering medication, mipomersen is associated with higher costs compared to other treatments. The average wholesale price for a 30-day supply is approximately USD 23,038.60. However, the cost-effectiveness of mipomersen has not been extensively evaluated.