Posts Tagged ‘familial hypercholesterolemia’

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October 6th, 2014

Selections from Richard Lehman’s Literature Review: October 6th

This week’s topics include medical device safety and effectiveness, familial hypercholesterolemia, and more.


February 21st, 2013

Study Casts Doubt on Value of Genetic Testing for Familial Hypercholesterolemia

The recent introduction of two drugs specifically targeted to treat people with the rare but dangerous condition of homozygous familial hypercholesterolemia (FH) has caused increased interest in figuring out the best strategy to identify people with the disorder. Now a new study published online in the Lancet suggests that one of the main screening plans that relies on genetic […]


December 26th, 2012

FDA Approves Lomitapide for Homozygous Familial Hypercholesterolemia

Aegerion Pharmaceuticals said today that the FDA had approved lomitapide (Juxtapid) to help further lower cholesterol in patients with homozygous familial hypercholesterolemia. The approval comes with a box warning about the risk of hepatotoxicity and a Risk Evaluation and Mitigation Strategy (REMS) Program which will require certification of health care providers and pharmacies before the drug can be […]


December 14th, 2012

CHMP Recommends Against Approval for Mipomersen in Europe

The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency today recommended that mipomersen (Kynamro; Isis and Genzyme) not be approved for use in Europe. The novel antisense oligonucleotide works by inhibiting the synthesis of apolipoprotein-B and is under development in the United States and Europe for the treatment of familial hypercholesterolemia. CHMP […]


October 18th, 2012

FDA Panel Recommends Approval of Mipomersen for Homozygous Familial Hypercholesterolemia

The FDA’s Endocrinologic and Metabolic Drugs Advisory Committee gave a weak endorsement to mipomersen, an antisense oligonucleotide inhibitor manufactured by Genzyme, for use in homozygous familial hypercholesterolemia (FH). With its relatively close 9-6 vote, and with its comments, the committee expressed concerns about both the efficacy and safety of the drug, but ultimately the severity of […]


October 18th, 2012

FDA Reviewers Recommend Approval of Lomitapide for Homozygous Familial Hypercholesterolemia

The FDA’s Endocrinologic and Metabolic Drugs Advisory Committee voted 13-2 on Wednesday to recommend approval of Aegerion Pharmaceuticals’ cholesterol-lowering drug lomitapide for use in patients with homozygous familial hypercholesterolemia (FH). The lopsided vote does not completely reflect the views of many of the panel members, who expressed considerable concern  that the drug might be used in […]


October 16th, 2012

FDA Review Raises Safety Concerns About Mipomersen

An FDA review raises a number of potentially significant safety concerns about the cholesterol-lowering drug mipomersen. The review appears ahead of a Thursday meeting of the Endocrinologic and Metabolic Drugs Advisory Committee to evaluate Genzyme’s new drug application (NDA) for use of the drug as an adjunct to maximally tolerated lipid-lowering medications and diet to reduce […]


October 15th, 2012

FDA Reviewers Raise No New Red Flags Over Lomitapide

FDA reviewers have raised no new concerns about lomitapide ahead of a Wednesday meeting of the Endocrinologic and Metabolic Drugs Advisory Committee.  The FDA today released briefing documents that evaluate the new drug application (NDA) for lomitapide capsules, the microsomal triglyceride transfer protein (MTP) inhibitor from Aegerion Pharmaceuticals. It’s intended for use as an adjunct to a […]


March 22nd, 2012

Promising Phase 1 Results for New Monoclonal Antibody to PCSK9

Promising results from very early studies with an experimental new cholesterol-lowering drug, a monoclonal antibody to PCSK9, have been published in the New England Journal of Medicine. Evan Stein and colleagues report the results of two single-dose studies in which the drug, REGN727, was administered intravenously or subcutaneously to healthy subjects. In a third, randomized, placebo-controlled, dose-ranging trial, […]