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Oct. 18, 2012

FDA Should Reject Dangerous Cholesterol Medication, Public Citizen Tells Advisory Committee

Mipomersen, Studied in an Unethical Clinical Trial, Shown to Cause a Plethora of Side Effects

WASHINGTON, D.C. – The U.S. Food and Drug Administration (FDA) should not approve the proposed cholesterol drug mipomersen because the study supporting approval was unethical and withheld an effective therapy from patients with a rare disease, Public Citizen said today in testimony to the FDA’s Endocrinologic and Metabolic Drugs Advisory Committee.

Mipomersen is the first of a new class of drugs developed to treat homozygous familial hypercholesterolemia (HoFH), an extremely rare, fatal genetic condition that leads to very high blood cholesterol levels.

However, Public Citizen testified that the pivotal trial conducted in HoFH patients to support the drug’s approval was unethical because investigators withheld an effective, standard, life-prolonging treatment – LDL apheresis – from all patients.

This and other trials also showed that mipomersen caused liver damage, flu-like symptoms and possibly cancer in a large number of patients, according to FDA reviewers. Mostly due to these side effects, more than half of the HoFH patients stopped taking mipomersen before the trial was completed. But because the drug stays in the bloodstream long after discontinuation, side effects continued in many HoFH and other patients for several months afterwards.

Of further concern, animal studies also demonstrated that the drug is carcinogenic at doses close to those that would be used in human patients.

“In the zeal to demonstrate mipomersen to be effective, the trial excluded the use of a known beneficial treatment, LDL apheresis,” said Dr. Sidney Wolfe, director of Public Citizen’s Health Research Group. “It will be interesting to find out how the institutional review boards overseeing this trial (or the FDA) allowed it to go forward. Until a more ethical trial is conducted, with more encouraging safety results, the drug should certainly not be approved.”

To read today’s testimony, please visit http://www.citizen.org/hrg2079.


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