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Mipomersen reduced atherosclerotic lipoproteins in familial hypercholesterolemia
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Mipomersen effectively and safely reduced all atherosclerotic lipoproteins associated with familial hypercholesterolemia for a sustained time period, according to recently published data.
In an open-label extension of the phase 3 study of mipomersen, researchers reported changes in LDL cholesterol and apolipoprotein B along with non-HDL cholesterol and total cholesterol from baseline through 104 weeks.
With weekly 200 mg subcutaneous injections of mipomersen added to maximum lipid-lowering therapy in 141 patients, LDL-C was reduced by 28% at 26 weeks (n=130), 27% at 52 weeks (n=111), 27% at 76 weeks (n=66) and 28% at 104 weeks (n=53). ApoB was reduced by 29% at 26 weeks, 28% at 52 weeks, 30% at 76 weeks and 31% at 104 weeks (P<.001).
Concurrently, mean non-HDL increased from baseline by 7% and 6% at 26 and 52 weeks, respectively.
“Mipomersen had a safety and tolerability profile consistent with that seen in the randomized placebo-controlled phase 3 studies. Longer term assessments of transaminase elevations and hepatic steatosis are needed, but the current data are reassuring,” researchers wrote. “Forthcoming data from the 4-year time point will help substantiate these findings.”
Researchers reported an “incremental increase” in medial liver fat through the first 6 to 12 months of therapy but the effect lessened with continued treatment with mipomersen for more than 1 year, returning to baseline 24 weeks after discontinuation.
All patients reported at least one treatment-emergent adverse event, with 98% reporting at least one injection site reaction and 65% reporting at least one episode of flu-like symptoms, which was the most commonly cited reason for discontinuing treatment.
Serious adverse events occurred in 23% of patients (n=33), though researchers attributed four to mipomersen.
Disclosure: This study was supported by the Genzyme Corporation. Please see study for full list of author disclosures.
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