–Novel strategy would identify both children and adults at high risk for CV events. Doctors in the U.K. are proposing a novel strategy to identify very young children with familial hypercholesterolemia (FH). The strategy would also identify and help prevent events in the parents who also have FH. The new approach begins with a heel-stick…
Not So Rare, FH Is Truly Risky Lipid Disorder
–Not all elevated is LDL is equally dangerous CHICAGO — Familial hypercholesterolemia (FH), a condition that has long been overshadowed by more common cardiovascular diagnoses, has now found the spotlight in the big tent as a hot and even controversial topic. The new interest is driven by important advances in understanding the genetics of the…
New Definition Of Familial Hypercholesterolemia Could Expand Patient Population For Expensive Cholesterol Drugs
More people may be diagnosed with familial hypercholesterolemia (FH) using criteria contained in a new scientific statement published by the American Heart Association. The expanded definition could also mean more patients will be eligible to receive expensive cholesterol-lowering drugs, including the new PCSK9 inhibitor drugs, (Repatha from Amgen and Praluent from Sanofi/Regeneron). But the biggest impact might be to increase the…
Praising The Virtues Of The Familial Hypercholesterolemia Foundation
The following letter was sent by Joshua Knowles in response to a previous post, Desperately Seeking Patients: New Cholesterol Drug Makers Fuel Research To Find Customers. Dr. Knowles is a faculty member and clinician at Stanford University who specializes in familial hypercholesterolemia. He is also the chief medical advisor for the FH Foundation. Dear Mr. Husten, I…
Desperately Seeking Patients: New Cholesterol Drug Makers Fuel Research To Find Customers
Everyone expects that the makers of the new PCSK9 inhibitor cholesterol lowering drugs are going to make billions and billions of dollars from these innovative new drugs. But before that can happen the companies that make the drugs will need to find the patients who will take the drug. To help find these patients a central strategy…
Aegerion Warns About Negative Impact Of DOJ Investigation
Earlier this week in its annual report Aegerion Pharmaceuticals provided an update on its ongoing problems with the FDA and the Department of Justice. As previously reported here and on The Street and on CNBC, the company landed in hot water with the FDA last year after its CEO made a series of off-label statements on the CNBC Fast Money show. (The company’s only product, Juxtapid…
Justice Department Investigating Aegerion Over Marketing Of New Cholesterol Drug
Aegerion Pharmaceuticals disclosed today that it is being investigated by the US Department of Justice over its marketing of Juxtapid (lomitapide). The drug was approved in December 2012 to help further lower cholesterol in patients with homozygous familial hypercholesterolemia. The drug costs $250,000 a year. The approval came with a box warning about the risk of hepatotoxicity and a strict Risk Evaluation…
Study Casts Doubt On Value Of Genetic Testing For Familial Hypercholesterolemia
A new study published online in the Lancet suggests that one of the main screening plans that relies on genetic tests will fail to identify a substantial portion of people with familial hypercholesterolemia. Investigators from the UK and Belgium analyzed DNA from several cohorts of FH and non-FH patients. Their chief finding was that a large percentage…
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 and subcutaneously to healthy subjects. In a third, randomized, placebo-controlled, dose-ranging…
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