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

The FDA today approved the cholesterol-lowering drug mipomersen (Kynamro)—the first major RNA therapeutic developed by Carlsbad, CA-based Isis Pharmaceuticals (NASDAQ: [[ticker: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: [[ticker: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.

Author: Bruce V. Bigelow

In Memoriam: Our dear friend Bruce V. Bigelow passed away on June 29, 2018. He was the editor of Xconomy San Diego from 2008 to 2018. Read more about his life and work here. Bruce Bigelow joined Xconomy from the business desk of the San Diego Union-Tribune. He was a member of the team of reporters who were awarded the 2006 Pulitzer Prize in National Reporting for uncovering bribes paid to San Diego Republican Rep. Randy “Duke” Cunningham in exchange for special legislation earmarks. He also shared a 2006 award for enterprise reporting from the Society of Business Editors and Writers for “In Harm’s Way,” an article about the extraordinary casualty rate among employees working in Iraq for San Diego’s Titan Corp. He has written extensively about the 2002 corporate accounting scandal at software goliath Peregrine Systems. He also was a Gerald Loeb Award finalist and National Headline Award winner for “The Toymaker,” a 14-part chronicle of a San Diego start-up company. He takes special satisfaction, though, that the series was included in the library for nonfiction narrative journalism at the Nieman Foundation for Journalism at Harvard University. Bigelow graduated from U.C. Berkeley in 1977 with a degree in English Literature and from the Columbia University Graduate School of Journalism in 1979. Before joining the Union-Tribune in 1990, he worked for the Associated Press in Los Angeles and The Kansas City Times.