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FDA OKs First-in-Class Drug From Isis for Rare Cholesterol Disorder

Xconomy San Diego — 

The FDA today approved the cholesterol-lowering drug mipomersen (Kynamro)—the first major RNA therapeutic developed by Carlsbad, CA-based Isis Pharmaceuticals (NASDAQ: ISIS). Isis has been developing its lead drug candidate under a 2008 partnership with Cambridge, MA-based Genzyme, now part of the French drug giant Sanofi (NYSE: SNY).

In a statement released late today, the FDA says it has approved mipomersen as a treatment (in addition to lipid-lowering medications and diet) for patients with a rare condition that leads to very high cholesterol levels in their blood. Patients with the inherited disorder are at a higher risk of cardiovascular disease—so high, in fact, that heart attacks and death often occur before age 30.

The FDA approval triggers a $25 million milestone payment to Isis from Genzyme under terms of their 2008 partnership. Genzyme will manufacture mipomersen.

In a joint statement from Isis and Genzyme, Isis Chairman and CEO Stanley Crooke describes the drug as “the culmination of two decades of work to create a new, more efficient drug technology platform. As evidenced by our robust pipeline, our antisense drug discovery technology is applicable to many different diseases, including the treatment of a chronic and rare disease.”

The FDA approved the use of mipomersen as a once-a-week injection to lower so-called “bad cholesterol” (LDL-C) in patients with homozygous familial hypercholesterolemia (HoFH), an inherited condition that affects roughly one out of a million Americans. Patients with HoFH lack the ability to remove “bad cholesterol” from their blood, causing abnormally high levels of circulating LDL-C.

The statement also quotes Genzyme CEO David Meeker as saying, “As the leader in treatments for rare diseases, we are pleased to bring our expertise to HoFH patients living with this serious condition to better help them manage their disease.”

The drug carries a warning because it is associated with liver enzyme abnormalities and accumulation of fat in the liver, which could lead to progressive liver disease with chronic use.

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