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Kynamro wins FDA approval for homozygous familial hypercholesterolemia
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The FDA has approved mipomersen sodium as an adjunct to lipid-lowering medications and a healthy diet to reduce LDL, apolipoprotein B, total cholesterol and non-HDL in patients with homozygous familial hypercholesterolemia, according to a press release.
Approval was based on results of a randomized, double blind, placebo-controlled, multicenter clinical trial of 51 patients aged 12 to 53 years with homozygous familial hypercholesterolemia who were already taking lipid-lowering medications. Mipomersen sodium (Kynamro, Genzyme/Isis Pharmaceuticals) further reduced LDL levels by 25% (mean reduction, 113 mg/dL from a treated baseline of 439 mg/dL) and reduced other endpoints for atherogenic particles, according to the release.
Mipomersen sodium was deemed safe based on pooled results from four phase 3 randomized, double blind, placebo-controlled trials involving 390 patients. Two-hundred and sixty-one patients received weekly subcutaneous injections of 200 mg and 129 patients received placebo for a median of 25 weeks. The most commonly reported adverse effects reported with mipomersen sodium were injection-site reactions (5%), elevated alanine aminotransferase (3.4%), flu-like symptoms (2.7%) elevated aspartate aminotransferase (2.3%) and abnormal liver function test (1.5%). Eighteen percent of patients assigned mipomersen sodium and 2% assigned placebo discontinued treatment due to adverse reactions.
Mipomersen sodium contains a Boxed Warning citing the risk of hepatic toxicity. Patients taking the medication should have liver enzyme testing before drug commencement and periodically thereafter. The drug will be available only through a Risk Evaluation and Mitigation Strategy (REMS), called the Kynamro REMS, to educate prescribers about risk for hepatotoxicity, the need for monitoring and to restrict access to therapy to patients with a clinical or laboratory diagnosis consistent with homozygous familial hypercholesterolemia, according to the company.
In October, the FDA’s Endocrinologic and Metabolic Drugs Advisory Committee voted 9-6 to recommend approval of mipomersen sodium for the treatment of homozygous familial hypercholesterolemia.
The safety and effectiveness of mipomersen sodium is unknown in patients with hypercholesterolemia who do not have homozygous familial hypercholesterolemia. The medication’s effect on CV morbidity and mortality is undetermined, according to the release.