Aegerion Warns About Negative Impact Of DOJ Investigation Reply

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 (lomitapide) is a cholesterol-lowering drug indicated for the rare condition of homozygous familial hypercholesterolemia. It sells for $250,000 a year.) Now the company reports that by running “a corrective advertisement on CNBC” and by reviewing additional promotional material  it believes it will be able to resolve its problems with the FDA.

But the DOJ investigation may prove to be a bigger and more serious problem….

Click here to read the full post on Forbes.

 

About these ads

Justice Department Investigating Aegerion Over Marketing Of New Cholesterol Drug Reply

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 and Mitigation Strategy (REMS) Program which requires certification of health care providers and pharmacies before the drug can be prescribed and dispensed.

Last November the company received a warning letter from the FDA after Aegerion’s CEO, Mark Beer, made comments on CNBC improperly promoting the drug.

Click here to read the full post on Forbes.

 

 

Study Casts Doubt On Value Of Genetic Testing For Familial Hypercholesterolemia 3

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 of FH patients did not have one of the single genetic mutations known to cause FH. Instead, these patients, termed “polygenic,” were found to have variants in multiple genes, each of which had a small LDL-raising effect, but when combined resulted in LDL levels similar to those in people with known FH mutations.

According to one cholesterol expert: 

The take home is that genetic screening is not a panacea and that screening based on fasting ipids makes most sense. Indeed, I never understood genetic screening for this disease anyway, since the phenotype (hypercholesterolemia) is what kills people, not the genotype, the phenotype is readily available with a blood test, the disease is not immediately fatal (It takes years to develop), and treatment is based on the numbers. It is like asking for a gene test fro sickle cell anemia. You can look at blood and make the diagnosis.

Click here to read the complete story on Forbes.

 

Promising Phase 1 Results For New Monoclonal Antibody to PCSK9 Reply

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 trial, REGN727 was administered subcutaneously on days 1, 29, and 43 in adults with and without familial hypercholesterolemia (FH).

REGN727 was well tolerated and no subjects stopped taking the drug due to adverse events. Five patients had brief elevations in creatine kinase over three times the upper limit of normal. The drug had a powerful LDL-lowering effect: in the dose-ranging study in subjects already receiving atorvastatin

  • the 50 mg dose caused a 39% reduction of LDL cholesterol to 77.5 mg/dl,
  • the 100 mg dose caused a 53% reduction of LDL cholesterol to 61.3 mg/dl, and
  • the 150 mg dose caused a  61% reduction of LDL cholesterol to 53.8 mg/dl.

In an accompanying editorial, Stephen Young and Loren Fong write that “at this point, the status of PCSK9 therapeutics appears to be full speed ahead.” But they also express caution, noting that much work remains to be done:
Click to continue reading…