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Genzyme's mipomersen wins key FDA panel vote for rare cases of HoFH

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Genzyme and its partners at Isis Pharmaceuticals ($ISIS) won a key endorsement today for mipomersen, an experimental therapy for homozygous familial hypercholesterolemia. But the tepid 9-6 outcome left the new drug application looking relatively weak compared with the 13-2 vote that greeted the rival drug lomitapide from Aegerion Pharmaceuticals ($AEGR) yesterday.

As Aegerion CEO Marc Beer told FierceBiotech today, not all patients with the rare cholesterol condition are likely to benefit from only one drug, leaving room for two therapies for the tiny patient population. And the notion that two would be better than one seemed to win the day for Genzyme with a majority of the agency's outside experts. 

"We need a toolkit. We need as many options as possible for these patients," one panel member remarked, according to a report in Forbes.

The FDA's review of mipomersen--to be called Kynamro if it makes its way to the commercial market--included a sharp focus on potential liver disease and malignant growths. But Genzyme, like Aegerion, is proposing a REMS program to carefully track each patient's response to the drug with regular liver checks to make sure anyone who is being harmed can be taken off the drug before it's too late.

HoFH is a genetic condition in which dangerously high LDL levels can trigger potentially lethal cardiovascular reactions. If Genzyme and Isis prevail along with Aegerion and both drugs are approved, analysts will be paying close attention to whether Aegerion can make a case for improved efficacy and safety that can win over a solid majority of the patient population and their doctors. Aegerion says it can market this drug by itself, though, while Genzyme--a unit of Sanofi ($SNY)--would have an advantage with a much larger marketing group and a global reach that dates back years.

- here's the story from Dow Jones
- get the report from Forbes

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