Genzyme/Isis On Track To Submit Cholesterol Drug Mipomersen In 1H 2011
This article was originally published in Pharmaceutical Approvals Monthly
Mipomersen, Genzyme/Isis Pharmaceuticals' second-generation antisense drug for severe high cholesterol, is four for four in terms of collecting positive outcomes in Phase III trials. But in addition to lowered cholesterol, patients in all of those studies had elevated liver enzymes, leaving open the question whether regulators will consider the side effect a manageable trade off for filling an unmet medical need
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Statins represent a high standard of care for managing high blood cholesterol, bolstered by a strong evidence base, but new targets like PCSK9 and CETP are nevertheless stealing the spotlight nowadays. Whether new drugs, if they make it to market, will be used beyond narrow, targeted population segments is uncertain and will depend on results from large-scale outcomes studies showing significant efficacy and, importantly, reassurance of a solid safety profile.
Sanofi’s Genzyme business unit and Isis submitted an NDA for mipomersen, a first-in-class cholesterol-lowering antisense drug, in an orphan population of high-risk patients with a rare genetic cardiovascular disease – with an assist from three Phase III clinical trials from other, larger, high-risk populations, to help inform regulators.
It’s been a long haul, but Sanofi’s Genzyme business unit and Isis have submitted their NDA for a drug that helps lower cholesterol levels in high-risk patients with a rare genetic cardiovascular disease that doesn’t respond well to statins.