Currently Enrolling Trials
Kynamro (mipomersen sodium) inhibits the ApoB-100 molecule, a protein that plays a pivotal role in the production of low-density lipoprotein (LDL). It reduces LDL-C by preventing the formation of atherogenic lipoproteins, the particles that carry cholesterol through the bloodstream.
Kynamro is specifically indicated as an adjunct to lipid-lowering medications and diet to reduce low density lipoprotein-cholesterol (LDL-C), apolipoprotein B (apo B), total cholesterol 31 (TC) and non-high density lipoprotein-cholesterol (non-HDL-C) in patients with homozygous familial hypercholesterolemia (HoFH).
Mechanism of Action
Mipomersen is an antisense oligonucleotide targeted to human messenger ribonucleic acid (mRNA) for apo B-100, the principal apolipoprotein of LDL and its metabolic precursor, VLDL. Mipomersen is complementary to the coding region of the mRNA for apo B-100 and binds by Watson and Crick base pairing. The hybridization of mipomersen to the cognate mRNA results in RNase H-mediated degradation of the cognate mRNA thus inhibiting translation of the apo B-100 protein.
Adverse events associated with the use of Kynamro may include, but are not limited to, the following:
- Injection site reactions
- Flu-like symptoms
- Elevations in serum transaminases
Kynamro is supplied as a solution for subcutaneous injection. The recommended dose is 200 milligrams (mg) once weekly.
Clinical Trial Results
FDA approval of Kynamro was based on a multinational, randomized, placebo-controlled, 26-week trial in 51 subjects with HoFH. Kynamro was given as 200 mg weekly subcutaneous injections, as an adjunct to lipid-lowering medications. The primary efficacy endpoint was percent change in LDL-C from baseline to week 28. At week 28, the mean and median percent changes in LDL-C from baseline were -25 percent.
For additional information regarding Kynamro or homozygous familial hypercholesterolemia, please visit the Kynamro web page.