Core evidence clinical impact summary for mipomersen for familial hypercholesterolemia (FH)

Outcome measure	Evidence	Implications
Disease-oriented evidence
LDL-cholesterol reduction	Substantial	In patients with homozygous or heterozygous FH LDL-cholesterol reduction of 25%–47% on top of maximally tolerated drug therapy
Lipoprotein(a) reduction	Substantial	Approximately 30% lipoprotein(a) reduction; Significant reduction of lipoprotein(a), not achievable with currently available drugs
Patient-oriented evidence
Injection site reactions	Substantial	Most patients (70–100%) describe injection site reaction
Flu-like symptoms	Moderate	Approximately 30% of patients describe flu-like symptoms (not significantly different from placebo)
Elevated LFTs	Moderate	6%–15% of patients have elevated LFTs
Economic evidence	Unknown	The drug has not yet been approved by US FDA or European agencies

Abbreviations: LDL, low-density lipoprotein; LFT, liver function test; FDA, Food and Drug Administration.